340,749 results match your criteria Gene Therapy & Molecular Biology [Journal]


Clinical Pharmacogenetics Implementation Consortium (CPIC) Guideline for CYP2B6 and Efavirenz-containing Antiretroviral Therapy.

Clin Pharmacol Ther 2019 Apr 21. Epub 2019 Apr 21.

Department of Internal Medicine, Meharry Medical College School of Medicine, Nashville, TN, USA.

The human immunodeficiency virus (HIV) type-1 non-nucleoside reverse transcriptase inhibitor, efavirenz, is widely used to treat HIV-1 infection. Efavirenz is predominantly metabolized into inactive metabolites by CYP2B6, and patients with certain CYP2B6 genetic variants may be at increased risk for adverse effects, particularly central nervous system toxicity and treatment discontinuation. We summarize the evidence from the literature and provide therapeutic recommendations for efavirenz prescribing based on CYP2B6 genotypes. Read More

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http://dx.doi.org/10.1002/cpt.1477DOI Listing

A novel RNA aptamer identifies plasma membrane ATP synthase beta subunit as an early marker and therapeutic target in aggressive cancer.

Breast Cancer Res Treat 2019 Apr 20. Epub 2019 Apr 20.

Department of Medicine, Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami, USA.

Purpose: Primary breast and prostate cancers can be cured, but metastatic disease cannot. Identifying cell factors that predict metastatic potential could guide both prognosis and treatment.

Methods: We used Cell-SELEX to screen an RNA aptamer library for differential binding to prostate cancer cell lines with high vs. Read More

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http://link.springer.com/10.1007/s10549-019-05174-3
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http://dx.doi.org/10.1007/s10549-019-05174-3DOI Listing
April 2019
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Spinal Cord Injury: Animal Models, Imaging Tools and the Treatment Strategies.

Neurochem Res 2019 Apr 20. Epub 2019 Apr 20.

Inserm, U-1192-Laboratoire Protéomique, Réponse Inflammatoire et Spectrométrie de Masse-PRISM, Université de Lille, 59000, Lille, France.

Spinal cord injury (SCI) often leads to irreversible neuro-degenerative changes with life-long consequences. While there is still no effective therapy available, the results of past research have led to improved quality of life for patients suffering from partial or permanent paralysis. In this review we focus on the need, importance and the scientific value of experimental animal models simulating SCI in humans. Read More

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http://dx.doi.org/10.1007/s11064-019-02800-wDOI Listing

Timely and large dose of clotting factor IX provides better joint wound healing after hemarthrosis in hemophilia B mice.

Int J Hematol 2019 Apr 20. Epub 2019 Apr 20.

Shanghai Key Laboratory of New Drug Design, School of Pharmacy, East China University of Science and Technology, Shanghai, China.

Bleeding into the joints represents the major morbidity of severe hemophilia and predisposes it to hemophilic arthropathy (HA). In a reproducible hemarthrosis mouse model, we found distinct changes in thrombin activity in joint tissue homogenate following exposure of the joint to blood in wide type (WT) and hemophilic B mice. Specifically, at early time points (4 h and 24 h) after hemarthrosis, thrombin activity in WT mice quickly peaked at 4 h, and returned to baseline after 1 week. Read More

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http://link.springer.com/10.1007/s12185-019-02639-5
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http://dx.doi.org/10.1007/s12185-019-02639-5DOI Listing
April 2019
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Rapid automatic identification of parameters of the Bergman Minimal Model in Sprague-Dawley rats with experimental diabetes for adaptive insulin delivery.

Comput Biol Med 2019 Apr 8;108:242-248. Epub 2019 Apr 8.

IEEE Engineering in Medicine and Biology, Mexico City, Mexico. Electronic address:

Glucose-Insulin regulation models can be used to individualize insulin therapy. However, the experimental techniques currently used to identify the appropriate parameter sets of an individual are expensive, time consuming, and very unpleasant for the patient. Since there is a wide range of intrapersonal parameter variability, the identified parameters in a laboratory setting (at rest) are not optimal for dynamic conditions of daily activities. Read More

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http://dx.doi.org/10.1016/j.compbiomed.2019.03.028DOI Listing

Minicircle DNA purification: Performance of chromatographic monoliths bearing lysine and cadaverine ligands.

J Chromatogr B Analyt Technol Biomed Life Sci 2019 Apr 14;1118-1119:7-16. Epub 2019 Apr 14.

CICS-UBI - Centro de Investigação em Ciências da Saúde, Universidade da Beira Interior, Av. Infante D. Henrique, 6200-506 Covilhã, Portugal. Electronic address:

Minicircle DNA (mcDNA) technology is in the vanguard of vectors designed for gene therapy, since the absence of prokaryotic sequences confers to mcDNA higher biosafety in comparison to other DNA vectors. However, the presence of other isoforms and non-recombined parental molecules hampers the isolation of supercoiled (sc) mcDNA with the chromatographic methods already established for plasmid purification. In this work, two monolithic supports were modified with lysine and its decarboxylated derivative, cadaverine, to explore their performance in the sc mcDNA purification. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S15700232183170
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http://dx.doi.org/10.1016/j.jchromb.2019.04.024DOI Listing
April 2019
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Untargeted gas chromatography-mass spectrometry-based metabolomics analysis of kidney and liver tissue from the Lewis Polycystic Kidney rat.

J Chromatogr B Analyt Technol Biomed Life Sci 2019 Apr 12;1118-1119:25-32. Epub 2019 Apr 12.

Separation Science and Metabolomics Laboratory, Murdoch University, 90 South Street, Murdoch, WA 6150, Australia; Metabolomics Australia, Western Australia Node, Murdoch University, 90 South Street, Murdoch, WA 6150, Australia.

Polycystic kidney disease (PKD) encompasses a spectrum of inherited disorders that lead to end-stage renal disease (ESRD). There is no cure for PKD and current treatment options are limited to renal replacement therapy and transplantation. A better understanding of the pathobiology of PKD is needed for the development of new, less invasive treatments. Read More

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http://dx.doi.org/10.1016/j.jchromb.2019.04.021DOI Listing

Epigenetic Inhibition Puts Target Antigen in the Crosshairs of CAR T Cells.

Mol Ther 2019 Apr 18. Epub 2019 Apr 18.

Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital, Houston, TX, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2019.04.007DOI Listing

Preclinical Evaluation of Allogeneic CAR T Cells Targeting BCMA for the Treatment of Multiple Myeloma.

Mol Ther 2019 Apr 8. Epub 2019 Apr 8.

Allogene Therapeutics, Inc., 210 E. Grand Avenue, South San Francisco, CA 94080, USA. Electronic address:

Clinical success of autologous CD19-directed chimeric antigen receptor T cells (CAR Ts) in acute lymphoblastic leukemia and non-Hodgkin lymphoma suggests that CAR Ts may be a promising therapy for hematological malignancies, including multiple myeloma. However, autologous CAR T therapies have limitations that may impact clinical use, including lengthy vein-to-vein time and manufacturing constraints. Allogeneic CAR T (AlloCAR T) therapies may overcome these innate limitations of autologous CAR T therapies. Read More

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http://dx.doi.org/10.1016/j.ymthe.2019.04.001DOI Listing

Insulin transcriptionally down-regulates carboxylesterases through pregnane X receptor in an Akt-dependent manner.

Toxicology 2019 Apr 18. Epub 2019 Apr 18.

Department of Pharmacology, School of Basic Medicine, Nanjing Medical University, Nanjing, Jiangsu 211166, China. Electronic address:

Insulin is a major therapy for diabetes, and therefore, its role and mechanisms in the regulation of drug-metabolizing enzymes (DMEs), is of clinical importance to facilitate the rational drug use. Carboxylesterases are regarded as one of the major determinants of the metabolism and disposition of various substrates through their actions in the liver and intestine, alterations in the activity of CESs enzymes are often important causes of drug interactions. Therefore, investigation on the mechanism of CESs regulation is significantly important. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S0300483X183056
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http://dx.doi.org/10.1016/j.tox.2019.04.008DOI Listing
April 2019
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Susceptibility of Pseudomonas aeruginosa and antimicrobial activity using PK/PD analysis: an 18-year surveillance study.

Enferm Infecc Microbiol Clin 2019 Apr 17. Epub 2019 Apr 17.

Pharmacokinetic, Nanotechnology and Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad 7, 01006 Vitoria-Gasteiz, Spain. Electronic address:

Introduction: We analysed the changes in the susceptibility of Pseudomonas aeruginosa to antimicrobials over an 18-year period (2000-2017) in order to evaluate the adequacy of the antimicrobial therapy against this organism in patients admitted in a tertiary Spanish hospital (excluding the intensive care unit). In addition, the antimicrobial activity was evaluated using pharmacokinetic/pharmacodynamic (PK/PD) criteria as a microbiological surveillance tool.

Methods: Susceptibility was studied according to the Clinical and Laboratory Standards Institute breakpoints. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S0213005X193014
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http://dx.doi.org/10.1016/j.eimc.2019.02.009DOI Listing
April 2019
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Electrical stimulation induces direct reprogramming of human dermal fibroblasts into hyaline chondrogenic cells.

Biochem Biophys Res Commun 2019 Apr 17. Epub 2019 Apr 17.

Department of Physical Therapy and Rehabilitation, College of Health Science, Eulji University, Seongnam, Republic of Korea. Electronic address:

The repair of articular cartilage needs a sufficient number of chondrocytes to replace the defect tissue. Direct reprogramming of fibroblasts into chondrocytes can provide a sufficient number of chondrocytes because fibroblasts can be expanded efficiently. Herein, we demonstrate for the first time that electrical stimulation can drive direct reprogramming of human dermal fibroblasts (HDFs) into hyaline chondrogenic cells. Read More

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http://dx.doi.org/10.1016/j.bbrc.2019.04.027DOI Listing

Imaging features of triple-negative breast cancers according to androgen receptor status.

Eur J Radiol 2019 May 21;114:167-174. Epub 2019 Mar 21.

Department of Diagnostic Radiology, Unit 1350, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030, USA. Electronic address:

Objective: Different molecular subtypes of triple-negative breast cancer (TNBC) have previously been identified through analysis of gene expression profiles. The luminal androgen receptor (LAR) subtype has been shown to have a lower rate of pathologic complete response to neoadjuvant chemotherapy than other TNBC subtypes. The purpose of this study was to determine if the imaging features of TNBCs differ by AR (androgen receptor) status, which is a surrogate immunohistochemical (IHC) marker for the chemoresistant LAR subtype of TNBC. Read More

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http://dx.doi.org/10.1016/j.ejrad.2019.03.017DOI Listing

The anti-angiogenic effect of tryptanthrin is mediated by the inhibition of apelin promoter activity and shortened mRNA half-life in human vascular endothelial cells.

Phytomedicine 2019 Feb 26;58:152879. Epub 2019 Feb 26.

Graduate Institute of Clinical Medical Sciences, College of Medicine, Chang Gung University, 259 Wen-Hwa 1st Road, Kwei-Shan, Taoyuan, Taiwan, ROC; Department of Physical Medicine and Rehabilitation, Chang Gung Memorial Hospital, Taoyuan, Taiwan, ROC. Electronic address:

Background: Anti-angiogenesis is an important strategy of psoriasis treatment, but the side effects of systemic agents remain difficult to overcome. Topical use of indigo naturalis ointment has been proved to improve the skin lesion of psoriasis effectively and safely and one of its major components, tryptanthrin, has been demonstrated to have anti-angiogenic effect. Apelin, which has been reported to act as an angiogenic factor that could stimulate the proliferation and migration of vascular endothelial cells and proved to be elevated in psoriasis patients, is a potential target of anti-angiogenic therapy. Read More

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http://dx.doi.org/10.1016/j.phymed.2019.152879DOI Listing
February 2019

Human Erythroblasts with c-Kit Activating Mutations Have Reduced Cell Culture Costs and Remain Capable of Terminal Maturation.

Exp Hematol 2019 Apr 17. Epub 2019 Apr 17.

Center for Pediatric Biomedical Research, Department of Pediatrics, University of Rochester, Rochester, NY. Electronic address:

A major barrier to the in vitro production of red blood cells for transfusion therapy is the cost of culture components, with cytokines making up greater than half of the culture costs. Cell culture cytokines also represent a major expense for in vitro studies of human erythropoiesis. HUDEP2 cells are an E6/E7 immortalized erythroblast line used for the in vitro study of human erythropoiesis. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S0301472X193013
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http://dx.doi.org/10.1016/j.exphem.2019.04.001DOI Listing
April 2019
4 Reads

Multifunctional drug carrier based on PEI derivatives loaded with small interfering RNA for therapy of liver cancer.

Int J Pharm 2019 Apr 17. Epub 2019 Apr 17.

Jilin University, School of Life Sciences, Changchun, Jilin, China. Electronic address:

Gene therapy strategies for liver cancer have broad application prospects but still lack a stable and efficient delivery vehicle. To overcome this obstacle, we designed a multifunctional gene delivery vector, sTPssOLP, which was based on oleylamine (OA)-modified disulfide-containing polyethylenimine (PEI) and incorporated into lipids to prepare a lipid nanoparticle. sTPssOLP consisted of the core of PEI derivative and cationic lipids bound to siRNA. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S03785173193031
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http://dx.doi.org/10.1016/j.ijpharm.2019.04.049DOI Listing
April 2019
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Targeted methods for molecular characterization of EGFR mutational profile in lung cancer Moroccan cohort.

Gene 2019 Apr 17. Epub 2019 Apr 17.

Laboratory of Genetics and Molecular Pathology, Faculty of Medicine and Pharmacy of Casablanca, University Hassan II, Casablanca 20250, Morocco.

The study of EGFR gene mutational profile in NSCLC patients has a special clinical significance in the selection of patients for tyrosine-kinase inhibitors therapy. From 2017, the targeted therapy started to be accessible in public sector in Morocco, thus, the implementation of techniques for the molecular characterization of EGFR mutations in the laboratories became a necessity. The aim of this study was to present targeted methods "ADx-ARMS technology and the Idylla™ system" for the identification of EGFR mutational profile, methods that can be implemented in our clinical laboratories for routine analysis instead of outsourcing analysis to other countries. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S03781119193039
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http://dx.doi.org/10.1016/j.gene.2019.04.044DOI Listing
April 2019
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CAR T-cell bioengineering: Single variable domain of heavy chain antibody targeted CARs.

Adv Drug Deliv Rev 2019 Apr 17. Epub 2019 Apr 17.

School of Pharmacy, The Faculty of Medical Sciences, King George VI Building, Newcastle University, Newcastle upon Tyne NE1 7RU, UK; Division of Stratified Medicine, Biomarkers & Therapeutics, Institute of Cellular Medicine, Newcastle University, Framlington Place, Newcastle upon Tyne NE2 4HH, UK. Electronic address:

Redirecting the recognition specificity of T lymphocytes to designated tumour cell surface antigens by transferring chimeric antigen receptor (CAR) genes is becoming an effective strategy to combat cancer. Today, CAR T-cell therapy has proven successful in the treatment of haematological malignancies and the first CD19 CAR T-cell products has already entered the market. This success is expanding CAR design for broader malignancies including solid tumours. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S0169409X193004
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http://dx.doi.org/10.1016/j.addr.2019.04.006DOI Listing
April 2019
4 Reads

Lymph node fine-needle cytology in the era of personalized medicine. Is there a role?

Cytopathology 2019 Apr 20. Epub 2019 Apr 20.

Universita degli Studi di Salerno, Medicine and Surgery, Fisciano, Campania, IT.

The 2016 WHO revised classification of lymphoma has sub-classified well defined entities and added a number of provisional entities on the basis of new knowledge on genetic, epigenetics and phenotypical data; prognostic and predictive features are also part of this classification. New knowledge on well-defined entities further enlightens the mechanisms of lymphomagenesis, which are more complex and multifactorial than once believed. Therapies are also more complex because traditional clinical trials have been integrated with new drugs and compounds with unique mechanisms of actions against distinct molecular targets. Read More

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https://onlinelibrary.wiley.com/doi/abs/10.1111/cyt.12708
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http://dx.doi.org/10.1111/cyt.12708DOI Listing
April 2019
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Disruption of the MBD2-NuRD complex but not MBD3-NuRD induces high level HbF expression in human erythroid cells.

Haematologica 2019 Apr 19. Epub 2019 Apr 19.

Massey Cancer Center, Virginia Commonwealth University, Richmond, USA;

As high fetal hemoglobin levels ameliorate the underlying pathophysiologic defects in sickle cell anemia and β-thalassemia, understanding the mechanisms that enforce silencing of fetal hemoglobin postnatally offers the promise of effective molecular therapy. Depletion of the Nucleosome Remodeling and Deacetylase complex member MBD2 causes a 10-20 fold increase in γ-globin gene expression in adult β-globin locus yeast artificial chromosome transgenic mice. To determine the effect of MBD2 depletion in human erythroid cells, genome editing technology was utilized to knockout MBD2 in Human Umbilical cord Derived Erythroid Progenitor-2 cells resulting in γ/γ+β mRNA levels of ~50% and ~40% fetal hemoglobin by high performance liquid chromatography. Read More

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http://dx.doi.org/10.3324/haematol.2018.210963DOI Listing

Rapid generation of multivirus-specific T lymphocytes for the prevention and treatment of respiratory viral infections.

Haematologica 2019 Apr 19. Epub 2019 Apr 19.

Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA

Respiratory tract infections due to community-acquired respiratory viruses including respiratory syncytial virus, Influenza, parainfluenza virus 3 and human metapneumovirus are detected in up to 40% of allogeneic hematopoietic stem cell transplant recipients in whom they cause severe symptoms including pneumonia and bronchiolitis and can be fatal. Given the lack of effective antivirals and the data from our group demonstrating that adoptively transferred ex vivo-expanded virus-specific T cells can be clinically beneficial for the treatment of both latent (Epstein-Barr virus, cytomegalovirus, BK virus, human herpesvirus 6) and lytic (adenovirus) viruses, we investigated the potential for extending this immunotherapeutic approach to respiratory viruses. We now describe a system that rapidly generates a single preparation of polyclonal (CD4+ and CD8+) virus-specific T cells reactive against 12 antigens derived from 4 viruses (respiratory syncytial virus, Influenza, parainfluenza virus 3 and human metapneumovirus) that commonly cause post-transplant morbidity and mortality. Read More

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http://dx.doi.org/10.3324/haematol.2018.206896DOI Listing

Arsenic trioxide inhibits EMT in hepatocellular carcinoma by promoting lncRNA MEG3 via PKM2.

Biochem Biophys Res Commun 2019 Apr 16. Epub 2019 Apr 16.

Department of Interventional Radiology, Zhongshan Hospital, Fudan University, China. Electronic address:

Hepatocellular carcinoma (HCC) presents a great burden for patients worldwide, and metastasis of HCC remains problematic. Arsenic trioxide is a traditional drug that has shown excellent efficacy when applied as cancer therapy. Our study explored the antimetastatic mechanism of arsenic trioxide in HCC. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S0006291X193072
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http://dx.doi.org/10.1016/j.bbrc.2019.04.081DOI Listing
April 2019
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In Vitro Anticancer Properties of Copper Metallodendrimers.

Biomolecules 2019 Apr 18;9(4). Epub 2019 Apr 18.

Department of General Biophysics, Faculty of Biology and Environmental Protection, University of Lodz, Pomorska 141/143, 90-236, Lodz, Poland.

Newly synthesized carbosilane copper dendrimers (CCD) with chloride and nitrate surface groups seem to be good candidates to be used as gene and drug carriers in anti-cancer therapy, due to their properties such as size and surface charge. Copper attached to the nanoparticles is an important element of many biological processes and recently their anti-cancer properties have been widely examined. Zeta size and potential, transmission electron microscopy (TEM), circular dichroism (CD), analysis of haemolytic activity, and fluorescence anisotropy techniques were used to characterize copper dendrimers. Read More

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http://dx.doi.org/10.3390/biom9040155DOI Listing
April 2019
2 Reads

MMP-9 Downregulation with Lipid Nanoparticles for Inhibiting Corneal Neovascularization by Gene Silencing.

Nanomaterials (Basel) 2019 Apr 18;9(4). Epub 2019 Apr 18.

Pharmacokinetic, Nanotechnology & Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad 7, 01015 Vitoria-Gasteiz, Spain.

Gene silencing targeting proangiogenic factors have been shown to be a useful strategy in the treatment of corneal neovascularization (CNV). Among interference RNA (RNAi) molecules, short-hairpin RNA (shRNA) is a plasmid-coded RNA able to down-regulate the expression of the desired gene. It is continuously produced in the host cell, inducing a durable gene silencing effect. Read More

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https://www.mdpi.com/2079-4991/9/4/631
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http://dx.doi.org/10.3390/nano9040631DOI Listing
April 2019
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Personalizing therapy for older adults with acute myeloid leukemia: Role of geriatric assessment and genetic profiling.

Authors:
Vijaya Raj Bhatt

Cancer Treat Rev 2019 Apr 11;75:52-61. Epub 2019 Apr 11.

Department of Internal Medicine, Division of Hematology-Oncology, University of Nebraska Medical Center, Omaha, NE, United States; Fred and Pamela Buffett Cancer Center, University of Nebraska Medical Center, Omaha, NE, United States. Electronic address:

Acute myeloid leukemia (AML) presents therapeutic challenges in older adults because of high-risk leukemia biology conferring chemoresistance, and poor functional status resulting in increased therapy-related toxicities. Recent FDA approval of 8 new drugs for AML has increased therapeutic armamentarium and also provides effective low-intensity treatment options. Rational therapy selection strategies that consider individual's risk of therapy-related toxicities and probability of disease control can maximize benefits of available treatments. Read More

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http://dx.doi.org/10.1016/j.ctrv.2019.04.001DOI Listing

Co-assembled Ca Alginate-Sulfate Nanoparticles for Intracellular Plasmid DNA Delivery.

Mol Ther Nucleic Acids 2019 Mar 28;16:378-390. Epub 2019 Mar 28.

Avram and Stella Goldstein-Goren Department of Biotechnology Engineering, Ben-Gurion University of the Negev, Beer-Sheva 8410501, Israel; Regenerative Medicine and Stem Cell (RMSC) Research Center, Ben-Gurion University of the Negev, Beer-Sheva 8410501, Israel; The Ilse Katz Institute for Nanoscale Science and Technology, Ben-Gurion University of the Negev, Beer-Sheva 8410501, Israel. Electronic address:

Successful gene therapy requires the development of suitable carriers for the selective and efficient delivery of genes to specific target cells, with minimal toxicity. In this work, we present a non-viral vector for gene delivery composed of biocompatible materials, CaCl, plasmid DNA and the semi-synthetic anionic biopolymer alginate sulfate (AlgS), which spontaneously co-assembled to form nanoparticles (NPs). The NPs were characterized with a slightly anionic surface charge (Zeta potential [ζ] = -14 mV), an average size of 270 nm, and their suspension was stable for several days with no aggregation. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S21622531193006
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http://dx.doi.org/10.1016/j.omtn.2019.03.006DOI Listing
March 2019
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Electroconvulsive therapy "corrects" the neural architecture of visuospatial memory: Implications for typical cognitive-affective functioning.

Neuroimage Clin 2019 Apr 5;23:101816. Epub 2019 Apr 5.

Rotman Research Institute, University of Toronto, Canada. Electronic address:

Although electroconvulsive therapy (ECT) is a widely used and effective treatment for refractory depression, the neural underpinnings of its therapeutic effects remain poorly understood. To address this issue, here, we focused on a core cognitive deficit associated with depression, which tends to be reliably ameliorated through ECT, specifically, the ability to learn visuospatial information. Thus, we pursued three goals. Read More

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http://dx.doi.org/10.1016/j.nicl.2019.101816DOI Listing
April 2019
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Intracerebral Delivery in Complex 3-D Arrays: The Intracerebral Microinjection Instrument (IMI).

World Neurosurg 2019 Apr 16. Epub 2019 Apr 16.

Affiliated BaYi Brain Hospital, Army General Hospital of PLA, Nan Men Cang No.5, Dong Cheng District, Beijing, China.

This video article describes and illustrates the function and application of the Intracerebral Microinjection Instrument (IMI). This newly developed technology allows delivery of therapeutics within the human brain in complex three-dimensional arrays using a single pass or minimal overlying penetrations through brain tissue. The IMI utilizes a delivery microcannula with a reduced diameter that minimizes local trauma and is capable of delivering precise volumes of therapeutics to discrete brain substructures. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S18788750193107
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http://dx.doi.org/10.1016/j.wneu.2019.04.081DOI Listing
April 2019
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Genetic factors and therapy outcomes in persistent developmental stuttering.

J Commun Disord 2019 Apr 5;80:11-17. Epub 2019 Apr 5.

National Institute on Deafness and Other Communication Disorders, National Institutes of Health, 35A Convent Drive, Bethesda, MD 20892, USA. Electronic address:

Purpose: We investigated whether outcomes of therapy for persistent developmental stuttering differ in individuals who carry a mutation in one of the known genes associated with stuttering compared to individuals without such mutations.

Method: We studied outcomes of an intensive fluency shaping-based therapy program in individuals with persistent developmental stuttering. We evaluated a cohort of 51 stuttering individuals with who carried a mutation in either the GNPTAB, GNPTG, NAGPA, or AP4E1 gene. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S00219924183016
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http://dx.doi.org/10.1016/j.jcomdis.2019.03.007DOI Listing
April 2019
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The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease.

Biochim Biophys Acta Mol Cell Biol Lipids 2019 Apr 16. Epub 2019 Apr 16.

Department of Biology, Barnard College-Columbia University, New York, NY 10027, United States of America. Electronic address:

Niemann-Pick type C (NP-C) disease is a rare and fatal neurodegenerative disease typified by aberrations in intracellular lipid transport. Cholesterol and other lipids accumulate in the late endosome/lysosome of all diseased cells thereby causing neuronal and visceral atrophy. A cure for NP-C remains elusive despite the extensive molecular advances emanating from the identification of the primary genetic defect in 1997. Read More

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http://dx.doi.org/10.1016/j.bbalip.2019.04.002DOI Listing
April 2019
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Genetic silencing of striatal CaV1.3 prevents and ameliorates levodopa dyskinesia.

Mov Disord 2019 Apr 19. Epub 2019 Apr 19.

Department of Translational Science & Molecular Medicine, College of Human Medicine, Michigan State University, Grand Rapids, MI, USA.

Background: Levodopa-induced dyskinesias are an often debilitating side effect of levodopa therapy in Parkinson's disease. Although up to 90% of individuals with PD develop this side effect, uniformly effective and well-tolerated antidyskinetic treatment remains a significant unmet need. The pathognomonic loss of striatal dopamine in PD results in dysregulation and disinhibition of striatal CaV1. Read More

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https://onlinelibrary.wiley.com/doi/abs/10.1002/mds.27695
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http://dx.doi.org/10.1002/mds.27695DOI Listing
April 2019
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Antifibrinolytic therapy for preventing oral bleeding in patients with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions.

Cochrane Database Syst Rev 2019 Apr 19;4:CD011385. Epub 2019 Apr 19.

Van Creveldkliniek / Department of Haematology, University Medical Centre Utrecht, Heidelberglaan 100, Utrecht, Netherlands, 3584CT.

Background: Minor oral surgery or dental extractions (oral or dental procedures) are widely performed and can be complicated by hazardous oral bleeding, especially in people with an inherited bleeding disorder such as haemophilia or Von Willebrand disease (VWD). The amount and severity of singular bleedings depend on disease-related factors, such as the severity of the haemophilia, both local and systemic patient factors (such as periodontal inflammation, vasculopathy or platelet dysfunction) and intervention-related factors (such as the type and number of teeth extracted or the dimension of the wound surface). Similar to local haemostatic measures and suturing, antifibrinolytic therapy is a cheap, safe and potentially effective treatment to prevent bleeding complications in individuals with bleeding disorders undergoing oral or dental procedures. Read More

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http://doi.wiley.com/10.1002/14651858.CD011385.pub3
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http://dx.doi.org/10.1002/14651858.CD011385.pub3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6474399PMC
April 2019
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Preclinical evaluation of EPHX2 inhibition as a novel treatment for inflammatory bowel disease.

PLoS One 2019 19;14(4):e0215033. Epub 2019 Apr 19.

Computational Biology, GlaxoSmithKline, Collegeville, Pennsylvania, United States of America.

Epoxyeicosatrienoic acids (EETs) are signaling lipids produced by cytochrome P450 epoxygenation of arachidonic acid, which are metabolized by EPHX2 (epoxide hydrolase 2, alias soluble epoxide hydrolase or sEH). EETs have pleiotropic effects, including anti-inflammatory activity. Using a Connectivity Map (CMAP) approach, we identified an inverse-correlation between an exemplar EPHX2 inhibitor (EPHX2i) compound response and an inflammatory bowel disease patient-derived signature. Read More

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0215033PLOS
April 2019
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Gene Delivery Using Lipoplexes and Polyplexes: Principles, Limitations and Solutions.

Crit Rev Eukaryot Gene Expr 2019 ;29(1):29-36

Neurogenic Inflammation Research Center, Mashhad University of Medical Sciences, Mashhad, Iran; Biotechnology Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran.

Gene therapy has attracted considerable attention for the treatment of genetic and acquired diseases. Successful gene therapy occurs when the therapeutic genes penetrate targeted cells and become available to the intracellular active site. Currently, a promising approach in gene delivery is the use of nonviral gene delivery vectors that lack immunogenicity but have low toxicity and potential tissue specificity. Read More

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http://dx.doi.org/10.1615/CritRevEukaryotGeneExpr.2018025132DOI Listing
January 2019

GdX/UBL4A-knockout mice resist collagen-induced arthritis by balancing the population of T1/T17 and regulatory T cells.

FASEB J 2019 Apr 19:fj201802217RR. Epub 2019 Apr 19.

State Key Laboratory of Membrane Biology, School of Medicine, Tsinghua University, Beijing, China.

Rheumatoid arthritis (RA) is an autoimmune disease associated with synovial hyperplasia and bone and cartilage destruction. T cells, notably T helper (T)-1 and T17 cells, play a critical role in the pathologic process of RA. However, it remains unclear how T1 and T17 cells are regulated during RA. Read More

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http://dx.doi.org/10.1096/fj.201802217RRDOI Listing

Sarcoidosis occurring during BRAF/MEK inhibitors is associated with paradoxical ERK activation in Erdheim-Chester patients.

J Eur Acad Dermatol Venereol 2019 Apr 19. Epub 2019 Apr 19.

Sorbonne Université, Assistance Publique-Hôpitaux de Paris, Hôpital de la Pitié-Salpêtrière, Service de Médecine Interne 2, Centre National de Référence Maladies Systémiques Rares, Lupus Systémique, Syndrome des Anticorps Antiphospholipides et Histiocytoses, Paris, 75013, France.

Almost 70% of patients with Erdheim-Chester disease (ECD), a rare non-Langerhans cell histiocytosis, harbor the somatic V600E mutation of the BRAF gene. BRAF and MEK inhibitors are dramatically efficacious for treating ECD, particularly in cases of life-threatening manifestations [1]. Sarcoidosis-like disorders have been described during treatments with targeted therapies for cancers [2]. Read More

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https://onlinelibrary.wiley.com/doi/abs/10.1111/jdv.15636
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http://dx.doi.org/10.1111/jdv.15636DOI Listing
April 2019
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MicroRNA-145 inhibits proliferation and promotes apoptosis of HepG2 cells by targeting ROCK1 through the ROCK1/NF-κB signaling pathway.

Eur Rev Med Pharmacol Sci 2019 Apr;23(7):2777-2785

Department of Traditional Chinese Medicine, JiNing No. 1 People's Hospital, JiNing, China.

Objective: Hepatocellular carcinoma (HCC) is a malignant cancer with a high fatality rate, and the expression of microRNA-145 (miR-145) is significantly low in HCC tissue. Therefore, the effect of miR-145 on HCC was explored.

Patients And Methods: Primary hepatocellular carcinoma samples and corresponding normal samples, and HepG2 cells were analyzed using flow cytometry, 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide (MTT) assay, Real-time quantitative reverse transcription-polymerase chain reaction, Western blotting, and dual-luciferase reporter assay. Read More

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http://www.europeanreview.org/article/17551
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http://dx.doi.org/10.26355/eurrev_201904_17551DOI Listing
April 2019
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Current Knowledge of Ipilimumab and its use in Treating Non-small Cell Lung cancer.

Expert Opin Biol Ther 2019 Apr 19. Epub 2019 Apr 19.

d Marlene and Stewart Greenebaum Comprehensive Cancer Center, University of Maryland School of Medicine , Baltimore , MD , USA.

Introduction: The systemic treatment of non-small cell lung cancer (NSCLC) has changed dramatically with the identification of actionable mutations and the use of targeted agents. Unfortunately, many tumors will acquire resistance and >75% of NSCLC cases lack for an actionable gene aberration. In this setting, immunotherapy rises as effective therapeutic where immune checkpoint inhibitors have entered or are entering the market in many neoplasms, including NSCLC. Read More

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https://www.tandfonline.com/doi/full/10.1080/14712598.2019.1
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http://dx.doi.org/10.1080/14712598.2019.1610380DOI Listing
April 2019
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Transmitted HIV-1 drug resistance in a treatment naïve cohort of recently infected individuals from Chennai, India.

AIDS Res Hum Retroviruses 2019 Apr 19. Epub 2019 Apr 19.

National Institute for Research in Tuberculosis, HIV/AIDS, Chennai, Tamil Nadu, India ;

A cross sectional study was undertaken to examine the prevalence and pattern of HIV drug resistance mutations among recently HIV-1 infected and antiretroviral therapy-naïve individuals from Chennai, South India. The HIV-1 Pol gene encompassing the Protease and Reverse Transcriptase (RT) regions were analysed from 53 ART -naïve HIV-1 infected individuals using an in-house method for identifying drug resistance mutations by genotyping. The overall prevalence of transmitted drug resistance (TDR) was found to be 11. Read More

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http://dx.doi.org/10.1089/AID.2019.0022DOI Listing

Super-resolution imaging of structure, molecular composition and stability of single oligonucleotide polyplexes.

Nano Lett 2019 Apr 19. Epub 2019 Apr 19.

The successful application of gene therapy relies on the development of safe and efficient delivery vectors. Cationic polymers like cell-penetrating peptides (CPPs) can condense genetic material into nanoscale particles - polyplexes - and induce cellular uptake. To this point, several aspects of the nanoscale structure of polyplexes have remained elusive due to the difficulty to visualize the molecular arrangement of the two components with nanometer resolution. Read More

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http://pubs.acs.org/doi/10.1021/acs.nanolett.8b04407
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http://dx.doi.org/10.1021/acs.nanolett.8b04407DOI Listing
April 2019
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Intelligent TAT-coupled anti-HER2 immunoliposomes knock downed MDR1 to produce chemosensitize phenotype of multidrug resistant carcinoma.

J Cell Physiol 2019 Apr 18. Epub 2019 Apr 18.

Department of Medical Biotechnology, School of Advanced Technologies, Shahrekord University of Medical Sciences, Shahrekord, Iran.

Gene therapy using biocompatible cationic liposomes is amongst promising approaches that decreases death from cancers. Here an invasive multidrug resistant cell model has been developed by lentiviral transfection. In parallel phospholipids have been covalently conjugated to TAT, MMP2, and Herceptin. Read More

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http://dx.doi.org/10.1002/jcp.28683DOI Listing

Overexpression miR-24-3p repressed Bim expression to confer tamoxifen resistance in breast cancer.

J Cell Biochem 2019 Apr 18. Epub 2019 Apr 18.

Department of Ultrasonic Diagnosis, the First Affiliated Hospital of China Medical University, Shenyang, China.

Endocrine therapy resistance represents a major challenge to the successful treatment of patients with breast cancer. The development of tamoxifen resistance commonly occurrs during the treatment of patients with breast cancer whereas its underlying mechanisms remain elusive. Here, we found that miR-24-3p regulated tamoxifen sensitivity in breast cancer cells. Read More

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http://dx.doi.org/10.1002/jcb.28568DOI Listing

Altered donor P2X7 activity in human leukocytes correlates with P2RX7 genotype but does not affect the development of graft-versus-host disease in humanised mice.

Purinergic Signal 2019 Apr 18. Epub 2019 Apr 18.

School of Chemistry and Molecular Bioscience, University of Wollongong, Wollongong, NSW, 2522, Australia.

Graft-versus-host disease (GVHD) is a life-threatening consequence of allogeneic haematopoietic stem cell transplantation, a curative therapy for haematological malignancies. The ATP-gated P2X7 receptor channel is implicated in the development of GVHD. P2X7 activity on human leukocytes can be influenced by gain-of-function (GOF) and loss-of-function (LOF) single nucleotide polymorphisms (SNPs) in the P2RX7 gene. Read More

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http://link.springer.com/10.1007/s11302-019-09651-8
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http://dx.doi.org/10.1007/s11302-019-09651-8DOI Listing
April 2019
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Biochemical Changes in Blood of Patients with Duchenne Muscular Dystrophy Treated with Granulocyte-Colony Stimulating Factor.

Biomed Res Int 2019 13;2019:4789101. Epub 2019 Mar 13.

Department of Pediatric Radiology, Medical University of Bialystok, Bialystok, Poland.

Introduction: In addition to the "gold standard" of therapy-steroids and gene therapy-there are experimental trials using granulocyte-colony stimulating factor (G-CSF) for patients with Duchenne muscular dystrophy (DMD). The aim of this study was to present the biochemical changes in blood after repeating cycles of granulocyte-colony stimulating factor G-CSF therapy in children with DMD.

Materials And Methods: Nineteen patients, aged 5 to 15 years, with diagnosed DMD confirmed by genetic tests, participated; nine were in wheelchairs, and ten were mobile and independent. Read More

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http://dx.doi.org/10.1155/2019/4789101DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6436375PMC

Genome-Wide and Phenotypic Evaluation of Stem Cell Progenitors Derived From -Deficient Murine Lung Adenocarcinoma With Somatic Mutations.

Front Oncol 2019 2;9:207. Epub 2019 Apr 2.

Department of Biochemistry and Molecular Genetics, Faculty of Medicine, American University of Beirut, Beirut, Lebanon.

Lung adenocarcinomas (LUADs) with somatic mutations in the oncogene comprise the most common molecular subtype of lung cancer in smokers and present with overall dismal prognosis and resistance to most therapies. Our group recently demonstrated that tobacco carcinogen-exposed mice with knockout of the airway lineage G-protein coupled receptor, , develop LUADs with somatic mutations in . Earlier work has suggested that cancer stem cells (CSCs) play crucial roles in clonal evolution of tumors and in therapy resistance. Read More

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http://dx.doi.org/10.3389/fonc.2019.00207DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6454871PMC

Orthopedic Manifestations of Bruck Syndrome: A Case Series with Intermediate to Long-term Follow-Up.

Case Rep Orthop 2019 13;2019:8014038. Epub 2019 Mar 13.

Department of Orthopedic Surgery, Alfred I. duPont Hospital for Children, Wilmington, DE, USA.

The aim of this study was to evaluate the association of contractures, fractures, and deformities in four patients with Bruck syndrome treated in our facility. Data were collected from medical records, radiographs, dual-energy X-ray absorptiometry (DEXA) scans, genetic tests, and gait analysis. All had contractures at birth and genotypic findings including mutations in PLOD2 or FPKB10. Read More

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http://dx.doi.org/10.1155/2019/8014038DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6436336PMC

Immunology's Coming of Age.

Front Immunol 2019 3;10:684. Epub 2019 Apr 3.

Department of Immunology, Max Planck Institute for Infection Biology, Berlin, Germany.

This treatise describes the development of immunology as a scientific discipline with a focus on its foundation. Toward the end of the nineteenth century, the study of immunology was founded with the discoveries of phagocytosis by Elias Metchnikoff, as well as by Emil Behring's and Paul Ehrlich's discovery of neutralizing antibodies. These seminal studies were followed by the discoveries of bacteriolysis by complement and of opsonization by antibodies, which provided first evidence for cooperation between acquired and innate immunity. Read More

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http://dx.doi.org/10.3389/fimmu.2019.00684DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6456699PMC
April 2019
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Roles of CircRNAs in Autoimmune Diseases.

Front Immunol 2019 2;10:639. Epub 2019 Apr 2.

Department of Laboratory Medicine, The Affiliated People's Hospital, Jiangsu University, Zhenjiang, China.

Circular RNAs (circRNAs) are covalently linked single-stranded RNAs, compared to linear counterparts that are relatively abundant, conserved, stable, and specific. Previously, most studies have revealed that circRNAs function in gene expression processes and participate in the pathogenesis of cancers, cardiovascular diseases, and neurological diseases. With advances in biotechnology, more biological functions of circRNAs have been found in several signaling pathways that are related to tumorigenesis, immunity, and metabolism. Read More

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https://www.frontiersin.org/article/10.3389/fimmu.2019.00639
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http://dx.doi.org/10.3389/fimmu.2019.00639DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6454857PMC
April 2019
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