780 results match your criteria Current Gene Therapy[Journal]


Gene-based therapeutic tools in the treatment of Cornea Disease.

Curr Gene Ther 2018 Dec 13. Epub 2018 Dec 13.

Tianjin Medical University Eye Hospital, The College of Optometry, Tianjin 300384. China.

Background: As one of the main blinding ocular diseases, corneal blindness results from neovascularization that disrupts the angiogenic privilege of corneal avascularity. Following neovascularization, inflammatory cells are infiltrating into corneal to strengthen corneal injury. How to maintain corneal angiogenic privilege to treat corneal disease has been investigated for decades. Read More

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http://dx.doi.org/10.2174/1566523219666181213120634DOI Listing
December 2018
2 Reads

Functional Improvement of Chimeric Antigen Receptor Through Intrinsic Interleukin-15Rα Signaling.

Curr Gene Ther 2018 Nov 15. Epub 2018 Nov 15.

Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida, 32610. United States.

Recent studies of CD19-specific chimeric antigen receptor (CAR)-modified T cells (CARTs) have demonstrated unprecedented successes in treating refractory and relapsed B cell malignancies. The key to the latest CART therapy advances can be attributed to the improved co-stimulatory signals in the CAR design. Here we established several novel CARs by incorporating T cell signaling domains of CD28 in conjunction with intracellular signaling motif of 4-1BB, CD27, OX40, ICOS, and IL-15Rα. Read More

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http://www.eurekaselect.com/167421/article
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http://dx.doi.org/10.2174/1566523218666181116093857DOI Listing
November 2018
25 Reads

Comparative Activity of Adenosine Deaminase Acting on RNA (ADARs) Isoforms for Correction of Genetic Code in Gene Therapy.

Curr Gene Ther 2018 Nov 14. Epub 2018 Nov 14.

School of Materials Science, Japan Advanced Institute of Science and Technology, 1-1 Asahidai, Nomi, Ishikawa 923-1292. Japan.

Members of the adenosine deaminase acting on RNA (ADAR) family of enzymes consist of double-stranded RNA-binding domains (dsRBDs) and a deaminase domain (DD) that converts adenosine (A) into inosine (I), which acts as guanosine (G) during translation. Using the MS2 system, we engineered the DD of ADAR1 to direct it to a specific target. The aim of this work was to compare the deaminase activities of ADAR1-DD and various isoforms of ADAR2-DD. Read More

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http://dx.doi.org/10.2174/1566523218666181114122116DOI Listing
November 2018
20 Reads

Design of Artificial Immunogens Containing Melanoma-associated T-cell Epitopes.

Curr Gene Ther 2018 ;18(6):375-385

State Research Center of Virology and Biotechnology "Vector", Koltsovo, Novosibirsk Region, Russian Federation.

Objective: Immunotherapy based on induction of T-cell responses is a promising approach to cancer treatment. The study aims to design artificial epitope-based immunogens, DNA vaccine candidates against melanoma and evaluate their ability to stimulate tumor cytotoxicity of ex vivo generated T-cells.

Methods: The original computational methods were used for predicting T-cell epitopes and designing polyepitope melanoma antigens. Read More

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http://www.eurekaselect.com/167302/article
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http://dx.doi.org/10.2174/1566523218666181113112829DOI Listing
January 2018
12 Reads

Emerging Therapeutic Approaches for Diamond Blackfan Anemia.

Curr Gene Ther 2018 ;18(6):327-335

Department of Health Sciences, University of Eastern Piedmont Amedeo Avogadro, Novara, Italy.

Diamond Blackfan Anemia (DBA) is an inherited erythroid aplasia with onset in childhood. Patients carry heterozygous mutations in one of 19 Ribosomal Protein (RP) genes, that lead to defective ribosome biogenesis and function. Standard treatments include steroids or blood transfusions but the only definitive cure is allogeneic Hematopoietic Stem Cell Transplantation (HSCT). Read More

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http://dx.doi.org/10.2174/1566523218666181109124538DOI Listing
January 2018
14 Reads

Clinical-Grade Oncolytic Adenovirus Purification Using Polysorbate 20 as an Alternative for Cell Lysis.

Curr Gene Ther 2018 ;18(6):366-374

iBET, Instituto de Biologia Experimental e Tecnologica, Oeiras, Portugal.

Introduction: Oncolytic virus therapy is currently considered as a promising therapeutic approach for cancer treatment. Adenovirus is well-known and extensively characterized as an oncolytic agent. The increasing number of clinical trials using this virus generates the demand for the development of a well-established purification approach. Read More

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http://dx.doi.org/10.2174/1566523218666181109141257DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6327138PMC
January 2018
7 Reads
2.542 Impact Factor

Circular RNAs Serve as Novel Biomarkers and Therapeutic Targets in Cancers.

Curr Gene Ther 2018 Nov 9. Epub 2018 Nov 9.

Department of Biochemistry and Molecular Biology, Medical School of Ningbo University, Ningbo. China.

Circular RNAs (circRNAs) are a class of non-coding RNAs (ncRNAs) that structurally form closed loops without 5'-end cap and 3'-end poly(A) tail unlike linear RNAs. CircRNAs are widely present in eukaryotic cells and are presented with capabilities and characteristics of structural stability, high abundance and cell-/tissue-specific expression. A growing body of research now suggests that the aberrant expression of circRNA is intimately relevant to the occurrence and development of cancer. Read More

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http://www.eurekaselect.com/167213/article
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http://dx.doi.org/10.2174/1566523218666181109142756DOI Listing
November 2018
6 Reads

Clinical Observation of Patients with Leber's Hereditary Optic Neuropathy Before Gene Therapy.

Curr Gene Ther 2018 ;18(6):386-392

Department of Ophthalmology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.

Background: Leber's hereditary optic neuropathy is a hereditary mitochondrial disease. No effective treatment has so far been established, with gene therapy currently being the most promising. Because of the possibility of spontaneous visual acuity recovery in this disease, we screened patients before gene therapy, excluding those with spontaneous visual acuity improvement, and prepared for the subsequent gene therapy. Read More

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http://dx.doi.org/10.2174/1566523218666181105125245DOI Listing
January 2018
45 Reads

Human Disease System Biology.

Authors:
Liang Cheng Yang Hu

Curr Gene Ther 2018 Nov 1. Epub 2018 Nov 1.

Department of Bioinformatics School of Life Science and Technology Harbin Institute of Technology, Harbin. China.

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http://dx.doi.org/10.2174/1566523218666181101143116DOI Listing
November 2018
7 Reads

ExomiRs: A Novel Strategy in Cancer Diagnosis and Therapy.

Curr Gene Ther 2018 ;18(6):336-350

Department of Pharmaceutics and Nanotechnology, School of Pharmacy, Shahid Beheshti University of Medical Sciences, Tehran, Iran.

Exosomes play a critical role in intercellular communication between cancer cells and their environments. These secreted nanovesicles can transfer different cargos such as mRNAs, proteins and microRNA (miRNA) to recipient cells. Exosomal miRNAs (exomiRs) derived from tumor cells have emerged as key players in cancer promotion via impairment of the immune system response, tumor growth, metastasis, angiogenesis, and chemotherapeutic drug resistance. Read More

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http://dx.doi.org/10.2174/1566523218666181017163204DOI Listing
January 2018
17 Reads

An Observation of the Role of Autophagy in Patients with Endometriosis of Different Stages during Secretory Phase and Proliferative Phase.

Curr Gene Ther 2018 ;18(5):286-295

Department of Gynecology and Obstetrics, the Second Affiliated Hospital of Harbin Medical University, Harbin, China.

Background: Autophagy exists widely in various physiological and pathological conditions. Lots of investigations have verified that the autophagic activity is always related to the occurrence and the development of cancer. Endometriosis (EMs) is a disease that endometrium-like tissues abnormally grow outside the uterus and also considered to possess the characters of tumor because of its malignant biological behavior. Read More

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http://dx.doi.org/10.2174/1566523218666181008155039DOI Listing
January 2018
2 Reads

Human Disease System Biology.

Authors:
Liang Cheng Yang Hu

Curr Gene Ther 2018 ;18(5):255-256

Department of Bioinformatics School of Life Science and Technology Harbin Institute of Technology, Harbin, China.

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http://dx.doi.org/10.2174/1566523218666181010101114DOI Listing
January 2018
7 Reads

Identifying Keystone Species in the Microbial Community Based on Cross- Sectional Data.

Curr Gene Ther 2018 ;18(5):296-306

School of Computer Science and Technology, Harbin Institute of Technology, WeiHai, China.

Background: In microbial communities, the keystone species have a greater impact on the performance and dynamics of ecosystem than that of other species, in which we can see from the results that losing gut microbiome causes some specific diseases. A number of ongoing studies aim at identifying links between microbial community structure and human diseases.

Method: In this paper, we have introduced a valid keystone species identification method, in which a new Spread Intensity (SI) algorithm is used. Read More

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http://www.eurekaselect.com/166055/article
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http://dx.doi.org/10.2174/1566523218666181008155734DOI Listing
January 2018
7 Reads
2.540 Impact Factor

EZH2 RIP-seq Identifies Tissue-specific Long Non-coding RNAs.

Curr Gene Ther 2018 ;18(5):275-285

Department of Cardiology, Central Laboratory, Renmin Hospital, Wuhan University, Wuhan 430060, China.

Background: Polycomb Repressive Complex 2 (PRC2) catalyzes histone methylation at H3 Lys27, and plays crucial roles during development and diseases in numerous systems. Its catalytic subunit EZH2 represents a key nuclear target for long non-coding RNAs (lncRNAs) that emerging to be a novel class of epigenetic regulator and participate in diverse cellular processes. LncRNAs are characterized by high tissue-specificity; however, little is known about the tissue profile of the EZH2- interacting lncRNAs. Read More

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http://www.eurekaselect.com/166028/article
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http://dx.doi.org/10.2174/1566523218666181008125010DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6249712PMC
January 2018
4 Reads

Advances in Gene Therapy for Erectile Dysfunction: Promises and Challenges.

Curr Gene Ther 2018 ;18(6):351-365

Andrology Laboratory, West China Hospital, Sichuan University, Chengdu 610041, China.

Erectile Dysfunction (ED) is one of the most common conditions affecting middle-aged and older men. Over the past few decades, oral phosphodiesterase type 5 inhibitors have been used to treat ED. However, these oral medications require on-demand access and are not effective in some hard-totreat populations. Read More

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http://dx.doi.org/10.2174/1566523218666181004145424DOI Listing
January 2018
2 Reads

Nanomedicine for gene delivery for the treatment of cardiovascular diseases.

Curr Gene Ther 2018 Oct 3. Epub 2018 Oct 3.

Beijing Key Laboratory of Diabetes Prevention and Research, Endocrinology Center, Lu He Hospital, Capital Medical University, Beijing, 101149. China.

Myocardial infarction (MI) is the most severe ischemic heart disease and directly leads to heart failure till death. Target molecules have been identified in the event of MI including increasing angiogenesis, promoting cardiomyocyte survival, improving heart function and restraining inflammation and myocyte activation and subsequent fibrosis. All of which are substantial in cardiomyocyte protection and preservation of cardiac function. Read More

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http://www.eurekaselect.com/165896/article
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http://dx.doi.org/10.2174/1566523218666181003125308DOI Listing
October 2018
34 Reads

Neuroprotection by Human Dental Pulp Mesenchymal Stem Cells: From Billions to Nano.

Curr Gene Ther 2018 ;18(5):307-323

School of Regenerative Medicine, Manipal Academy of Higher Education, Bangalore, Karnataka, India.

Introduction: Mesenchymal Stem Cell (MSC) therapy in recent years has gained significant attention. Though the functional outcomes following MSC therapy for neurodegenerative diseases are convincing, various mechanisms for the functional recovery are being debated. Nevertheless, recent studies convincingly demonstrated that recovery following MSC therapy could be reiterated with MSC secretome per se thereby shifting the dogma from cell therapy to cell "based" therapy. Read More

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http://dx.doi.org/10.2174/1566523218666180913152615DOI Listing
January 2018
4 Reads

Deep Forest-based Prediction of Protein Subcellular Localization.

Curr Gene Ther 2018 ;18(5):268-274

Department of Rehabilitation, Heilongjiang Province Land Reclamation Headquarters General Hospital, Harbin, China.

Motivation: Knowledge of the correct protein subcellular localization is necessary for understanding the function of a protein and revealing the mechanism of many human diseases due to protein subcellular mislocalization, which is required before approaching gene therapy to treat a disease. In addition, it is well-known that the gene therapy is an effective way to overcome disease by targeting a gene therapy product to a specific subcellular compartment. Deep neural networks to predict protein function have become increasingly popular due to large increases in the available genomics data due to its strong superiority in the non-linear classification ability. Read More

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http://dx.doi.org/10.2174/1566523218666180913110949DOI Listing
January 2018
4 Reads

A Brief Survey of Machine Learning Application in Cancerlectin Identification.

Curr Gene Ther 2018 ;18(5):257-267

Key Laboratory for Neuro-Information of Ministry of Education, School of Life Science and Technology, Center for Informational Biology, University of Electronic Science and Technology of China, Chengdu 610054, China.

Proteins with at least one carbohydrate recognition domain are lectins that can identify and reversibly interact with glycan moiety of glycoconjugates or a soluble carbohydrate. It has been proved that lectins can play various vital roles in mediating signal transduction, cell-cell recognition and interaction, immune defense, and so on. Most organisms can synthesize and secret lectins. Read More

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http://dx.doi.org/10.2174/1566523218666180913112751DOI Listing
January 2018
34 Reads

Oncolytic Virotherapy for Breast Cancer Treatment.

Curr Gene Ther 2018 ;18(4):192-205

Department of Comparative Biomedical Sciences, School of Veterinary Medicine, Louisiana State University, Baton Rouge, LA, United States.

Breast cancer continues to be a leading cause of mortality among women. While at an early stage, localized breast cancer is easily treated; however, advanced stages of disease continue to carry a high mortality rate. The discrepancy in treatment success highlights that current treatments are insufficient to treat advanced-stage breast cancer. Read More

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http://dx.doi.org/10.2174/1566523218666180910163805DOI Listing
January 2018
19 Reads

Cellular, Molecular and Non-Pharmacological Therapeutic Advances for the Treatment of Parkinson's Disease: Separating Hope from Hype.

Curr Gene Ther 2018 ;18(4):206-224

Neuroscience Research Center, Baqiyatallah University of Medical Sciences, Tehran, Iran.

Parkinson's Disease (PD) is a frustrating condition characterized by motor and nonmotor deficits majorly caused by the loss of dopaminergic cells in the Substantia Nigra pars compacta (SNc) and destruction of the nigrostriatal pathway. Despite the very respectable advances in cutting-edge approaches for the treatment of PD, there exist numerous challenges that have incapacitated the definitive treatment of this disease. This review emphasized the development of various non-pharmaceutical therapeutic approaches and mainly highlighted the cutting-edge treatments for PD including gene- and stem cell-based therapies, targeted delivery of neurotrophic factors, and brain stimulation techniques such as Transcranial Magnetic Stimulation (TMS), transcranial Direct Current Stimulation (tDCS), and Deep Brain Stimulation (DBS). Read More

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http://dx.doi.org/10.2174/1566523218666180910163401DOI Listing
January 2018
1 Read

IGF-1 Gene Therapy as a Potentially Useful Therapy for Spontaneous Prolactinomas in Senile Rats.

Curr Gene Ther 2018 ;18(4):240-245

INIBIOLP-Pathology B, UNLP, La Plata, Argentina.

Background: Insulin-like Growth Factor1 (IGF1) is a powerful neuroprotective molecule. We have previously shown that short-term hypothalamic IGF1 gene therapy restores tuberoinfundibular dopaminergic neuron function in aging female rats.

Objective: Our aim was to implement long-term IGF-I gene therapy in pituitary prolactinomas in senile female rats. Read More

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http://dx.doi.org/10.2174/1566523218666180905170020DOI Listing
January 2018
2 Reads

Destination Penis? Gene Therapy as a Possible Treatment for Erectile Dysfunction.

Curr Gene Ther 2018 ;18(4):225-239

Department of Urology, Tulane University Health Sciences Center, New Orleans, LA, United States.

Erectile Dysfunction (ED) is a common health condition occuring in roughly 50% of aging males (40-70 years old). Recent attention has related gene therapy to ED, and now there is an interest to further implement gene therapy concepts to ED treatment. This review is an attempt to analyze key challenges and emphasize primary areas, including mostly preclinical and a few clinical trials, cellular target(s), and different viral vectors/nanoparticles for gene delivery in ED. Read More

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http://dx.doi.org/10.2174/1566523218666180730110432DOI Listing
January 2018
9 Reads

Phenotypic Characterization of a Family With An In-frame Deletion in the DMD Gene and Variable Penetrance.

Curr Gene Ther 2018 ;18(4):246-251

Inherited Cardiac Disease Unit, University Hospital Virgen de la Arrixaca, Murcia, Spain.

Duchenne muscular dystrophy is a disorder with variable expression caused by framedisrupting mutations in the dystrophin gene. It is characterized by progressive muscle weakness and dilated cardiomyopathy. In-frame dystrophin mutations cause a clinically moderate disorder named Becker muscular dystrophy. Read More

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http://www.eurekaselect.com/163634/article
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http://dx.doi.org/10.2174/1566523218666180709125346DOI Listing
January 2018
5 Reads

Current Advances in Small Activating RNAs for Gene Therapy: Principles, Applications and Challenges.

Curr Gene Ther 2018 ;18(3):134-142

State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center of Biotherapy, Chengdu 610041, China.

Small activating RNAs (saRNAs) are small double-stranded RNAs that could mediate the target-specific gene expression by targeting selected sequences in gene promoters at both the transcriptional and epigenetic levels. This phenomenon of gene manipulation is known as RNA activation (RNAa), which opens up a new pathway for RNA-based gene therapeutics in contrast to RNA interference. Although the exact molecular mechanism of RNAa mediated by saRNAs still remains foggy, some studies have provided the possible ones to explain it. Read More

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http://dx.doi.org/10.2174/1566523218666180619155018DOI Listing
January 2018
2 Reads

Inhibition of West Nile virus Replication by Bifunctional siRNA Targeting the NS2A and NS5 Conserved Region.

Curr Gene Ther 2018 ;18(3):180-190

Division of Virology, Defence Research and Development Establishment, Jhansi Road, Gwalior-474002, India.

Background: The West Nile Virus (WNV) has emerged as one of the most significant arboviral infection in many parts of the world and is associated with the encephalitis affecting mainly human and horses. In spite of the fact that the WNV is threat for the public health, there is no vaccine or therapeutic available for the treatment of WNV.

Methods: In this study, we tested a novel RNA interference based technique to inhibit WNV replication in Vero cells. Read More

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http://dx.doi.org/10.2174/1566523218666180607091311DOI Listing
January 2018
23 Reads

Editorial: Current Gene Therapy on Non-viral Gene Delivery and Therapy.

Authors:
Lihua Peng

Curr Gene Ther 2018 ;18(1)

College of Pharmaceutical Sciences, Zhejiang University Zhejiang Sheng, China.

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http://dx.doi.org/10.2174/156652321801180503164035DOI Listing
April 2019
1 Read

Preface: Translational Gene Therapy Coming of Age!

Curr Gene Ther 2018 ;18(1)

College of Medicine, University of Florida Gainesville, FL, United States.

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http://dx.doi.org/10.2174/156652321801180503163908DOI Listing
April 2019
1 Read

AAV-mediated Overexpression of IL-10 Mitigates the Inflammatory Cascade in Stimulated Equine Chondrocyte Pellets.

Curr Gene Ther 2018 ;18(3):171-179

Department of Clinical Sciences, Cornell University, Ithaca, NY 14853, United States.

Background: Following joint trauma, a posttraumatic inflammatory cascade drives degeneration of the joint. We aimed to assess whether transduction of chondrocytes with AAV5 overexpressing the immunomodulatory cytokine IL-10 would have protective effects in pellet cultures stimulated with IL-1β.

Methods: Chondrocytes were isolated from 3 healthy horses and were transduced with AAV5-IL-10 at a dose of 1 x 105vg/cell. Read More

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http://dx.doi.org/10.2174/1566523218666180510165123DOI Listing
January 2018
7 Reads

Editorial: Genetics and Gene Therapy of Lysosomial Storage Disorders.

Curr Gene Ther 2018 ;18(2):66-67

U.O.C.di Medicina Interna con Stroke Care Dipartimento Biomedico di Medicina Interna e Specialistica (Di.Bi.M.I.S) Universita degli Studi di Palermo Piazza delle Cliniche n.2, 90127, Palermo, Italy.

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http://dx.doi.org/10.2174/156652321802180507170539DOI Listing
January 2018
1 Read

miR-7 Replacement Therapy in Parkinson's Disease.

Curr Gene Ther 2018 ;18(3):143-153

Technology for Gene Therapy Laboratory, Central Institute of Sciences, FAV, University of Brasilia, Brasília, 70910- 900, Brazil.

The present review examines whether the microRNA 7 (miR-7) holds potential for slowing Parkinson's disease (PD) progression. First, the accurate expression of miR-7 allows for normal development, physiology, and neurogenesis in the central nervous system, also keeping alpha-synuclein (α-Syn) at the physiological level. Second, patients with PD and parkinsonian MPTP-induced animals exhibit a significant decrease of miR-7 in brain areas associated with dopaminergic neurodegeneration. Read More

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http://dx.doi.org/10.2174/1566523218666180430121323DOI Listing
January 2018
4 Reads

Transplantation of BDNF Gene Recombinant Mesenchymal Stem Cells and Adhesive Peptide-modified Hydrogel Scaffold for Spinal Cord Repair.

Curr Gene Ther 2018 ;18(1):29-39

Institute of Pharmaceutics, College of Pharmaceutical Sciences, Zhejiang University, Hangzhou, Zhejiang, China.

Introduction: Mesenchymal Stem Cells (MSCs) are promising candidates for nerve tissue engineering. Brain Derived Neurotrophic Factor (BDNF) secreted by MSCs can function to increase neural differentiation and relieve inflammation response. Gene transfection technology is an efficient strategy to increase the secretion levels of cytokines and enhance cellular functions. Read More

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http://dx.doi.org/10.2174/1566523218666180413150023DOI Listing
April 2019
4 Reads

Gene Therapy to Enhance Bone and Cartilage Repair in Orthopaedic Surgery.

Curr Gene Ther 2018 ;18(3):154-170

Department of Orthopaedic Surgery, Keck School of Medicine, University of Southern California, Los Angeles, CA, United States.

Musculoskeletal conditions are a major public health problem. Approximately 66 million individuals seek medical attention for a musculoskeletal injury in the United States, with current medical costs being estimated at $873 billion annually. Despite advances in pharmaceuticals, implant materials and surgical techniques, there remains an unmet clinical need for successful treatment of challenging musculoskeletal injuries and pathologic conditions, particularly in the setting of compromised biological environments. Read More

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http://dx.doi.org/10.2174/1566523218666180410152842DOI Listing
January 2018
42 Reads

Genetics and Gene Therapy in Hunter Disease.

Curr Gene Ther 2018 ;18(2):90-95

Department of Medical and Surgical Sciences, Pediatrics Unit, University "Magna Graecia", Catanzaro, Italy.

Mucopolysaccharidosis type II or Hunter syndrome is an X-linked lysosomal storage disease caused by a mutation in the gene encoding the lysosomal enzyme iduronate-2-sulfatase. The consequent enzyme deficiency causes a progressive, multisystem accumulation of glycosaminoglycans, which is the cause of the clinical manifestations involving also Central Nervous System for patients with the severe form of disease. The limits of the currently available therapies for Hunter syndrome, hematopoietic stem cell transplantation and recombinant enzyme replacement therapy, mainly regarding brain achievement, have encouraged several studies which recognized gene therapy as a potential therapeutic option for this condition. Read More

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http://dx.doi.org/10.2174/1566523218666180404155759DOI Listing
January 2018
7 Reads

Genetics and Gene Therapy of Anderson-Fabry Disease.

Curr Gene Ther 2018 ;18(2):96-106

Dipartimento Biomedico di Medicina Interna e Specialistica (Di.Bi.M.I.S), U.O.C.di Medicina Interna con Stroke Care, Università degli Studi di Palermo, Piazza delle Cliniche n.2, 90127, Palermo, Italy.

Fabry's disease is a genetic disorder of X-linked inheritance caused by mutations in the alpha galactosidase A gene resulting in deficiency of this lysosomal enzyme. The progressive accumulation of glycosphingolipids, caused by the inadequate enzymatic activity, is responsible of organ dysfunction and thus of clinical manifestations. In the presence of a high clinical suspicion, a careful physical examination and specific laboratory tests are required, finally diagnosis of Fabry's disease is confirmed by the demonstration of absence or reduced alpha-galactosidase A enzyme activity in hemizygous men and gene typing in heterozygous females; in fact the performance of enzymatic activity assay alone in women is inconclusive. Read More

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http://dx.doi.org/10.2174/1566523218666180404161315DOI Listing
January 2018
4 Reads

Genetics and Therapies for GM2 Gangliosidosis.

Curr Gene Ther 2018 ;18(2):68-89

Department of Medicine, University of Cambridge, Cambridge, United Kingdom.

Tay-Sachs disease, caused by impaired β-N-acetylhexosaminidase activity, was the first GM2 gangliosidosis to be studied and one of the most severe and earliest lysosomal diseases to be described. The condition, associated with the pathological build-up of GM2 ganglioside, has acquired almost iconic status and serves as a paradigm in the study of lysosomal storage diseases. Inherited as a classical autosomal recessive disorder, this global disease of the nervous system induces developmental arrest with regression of attained milestones; neurodegeneration progresses rapidly to cause premature death in young children. Read More

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http://dx.doi.org/10.2174/1566523218666180404162622DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6040173PMC
January 2018
8 Reads

Safety and Efficacy of Adenovirus Carrying Hepatocyte Growth Factor Gene by Percutaneous Endocardial Injection for Treating Post-infarct Heart Failure: A Phase IIa Clinical Trial.

Curr Gene Ther 2018 ;18(2):125-130

Department of Cardiology, the First Affiliated Hospital of Nanjing Medical University, Nanjing, Jiangsu, China.

Objective: Our previous phase I clinical trial has confirmed the safety of Adenovirus carrying Hepatocyte Growth Factor gene (Ad-HGF) by intracoronary administration for treating severe coronary artery disease. This study was performed to evaluate the safety and efficacy of Ad-HGF by percutaneous endocardial injection for treating post-infarct heart failure.

Methods: A total of 30 patients (15 in the experimental group and 15 in the control group) with postinfarct heart failure who were not indicated to revascularization and had received the optimal standardized medication therapy were included in the study. Read More

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http://dx.doi.org/10.2174/1566523218666180404162209DOI Listing
January 2018
19 Reads

Application of Optogenetics in Gene Therapy.

Curr Gene Ther 2018 ;18(1):40-44

Department of Medical Engineering, National Defense Medical College, 3-2 Namiki, Tokorozawa, Saitama, Japan.

The optogenetics approach uses a combination of genetic and optical methods to initiate and control functions in specific cells of biological tissues. Since the high-speed control of neuronal activity by irradiating channelrhodopsin-2 with blue light was reported in 2005, tremendous advancement and application of optogenetics in the field of neuroscience, such as in studies that associate neuronal activity with behaviors, have been initiated. Optogenetics is not only used as a research tool, but is also started to apply in the diagnosis of a disease or as therapy in various studies. Read More

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http://dx.doi.org/10.2174/1566523218666180302163814DOI Listing
April 2019
3 Reads

Dysfunction in Brain-Derived Neurotrophic Factor Signaling Pathway and Susceptibility to Schizophrenia, Parkinson's and Alzheimer's Diseases.

Curr Gene Ther 2018 ;18(1):45-63

Students` Scientific Research Center (SSRC), Tehran University of Medical Sciences, Tehran, Iran.

Brain-Derived Neurotrophic Factor (BDNF) is a dominant neurotrophic factor in the brain which plays a crucial role in differentiation, regeneration and plasticity mechanisms. Binding of the BDNF to its high-affinity Tropomyosin-related kinase B (TrkB) receptor leads to phosphorylation of TrkB, thus activating the three important downstream intracellular signaling cascades within the neural cells including phosphatidylinositol 3-kinase/protein kinase B (PI3K/AKT), Phospholipase C-γ (PLCγ), and mitogen-activated protein kinase/extracellular signal-related kinase (MAPK/ERK) pathways. Transcription of these pathways is regulated by cAMP Response Element-Binding protein (CREB) transcription factor, which can upregulate gene expression. Read More

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http://dx.doi.org/10.2174/1566523218666180302163029DOI Listing
April 2019
2 Reads

Applications and Prospects of Non-viral Vectors in Bone Regeneration.

Curr Gene Ther 2018 ;18(1):21-28

Key Laboratory of Biomaterials of Guangdong Higher Education Institutes, Department of Biomedical Engineering, Jinan University, Guangzhou 510632, China.

Introduction: Bone tissue has an intrinsic ability to repair and regenerate itself through a continuous remodelling cycle of resorption of old or damaged bone and deposition of new. However, significant morbidity and mortality arise when bone cannot heal itself. Effective bone regeneration strategy can improve the current clinical therapies of many orthopaedic disorders. Read More

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http://dx.doi.org/10.2174/1566523218666180227154232DOI Listing
April 2019
2 Reads

CRISPR-cas System as a Genome Engineering Platform: Applications in Biomedicine and Biotechnology.

Authors:
Atieh Hashemi

Curr Gene Ther 2018 ;18(2):115-124

Department of Pharmaceutical Biotechnology, School of Pharmacy, Shahid Beheshti University of Medical Sciences, Tehran, Iran.

Genome editing mediated by Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and its associated proteins (Cas) has recently been considered to be used as efficient, rapid and site-specific tool in the modification of endogenous genes in biomedically important cell types and whole organisms. It has become a predictable and precise method of choice for genome engineering by specifying a 20-nt targeting sequence within its guide RNA. Firstly, this review aims to describe the biology of CRISPR system. Read More

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http://dx.doi.org/10.2174/1566523218666180221110627DOI Listing
January 2018
30 Reads

What´s new in Gene Therapy of Hemophilia.

Curr Gene Ther 2018 ;18(2):107-114

Department of Orthopaedic Surgery, La Paz University Hospital, Madrid, Spain.

Background: Several methods have been investigated to effectively and safely transmit genes that stimulate cells to release therapeutic factor VIII (FVIII) and factor IX (FIX) into the circulation of people with hemophilia (PWH).

Objective: To review the role of gene therapy (GT) in PWH.

Methods: A Cochrane Library and PubMed (MEDLINE) search related to the role of GT in hemophilia was analyzed. Read More

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http://dx.doi.org/10.2174/1566523218666180214162312DOI Listing
January 2018
3 Reads

Role of Immune Cells in Diabetic Kidney Disease.

Curr Gene Ther 2017 ;17(6):424-433

Department of Nephrology, Ren Ji Hospital, School of Medicine, Shanghai Jiao Tong University, 160 Pujian Road, Shanghai 200127, China.

Diabetic Kidney Disease (DKD) is one of the major complications of Diabetes Mellitus (DM) and is currently the most common cause of End-Stage Renal Disease (ESRD) worldwide. Traditionally, DKD is considered a disease which has nothing to do with the immune system, and the pathogenesis is mainly characterized to be metabolic disturbance. Recent growing evidence indicates immunologic and inflammatory mechanisms in the development and progression of DKD. Read More

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http://dx.doi.org/10.2174/1566523218666180214100351DOI Listing
April 2019
2 Reads

Role of Complement Properdin in Renal Ischemia-Reperfusion Injury.

Curr Gene Ther 2017 ;17(6):411-423

Department of Infection, Immunity and Inflammation, College of Life Sciences, University of Leicester, Leicester, United Kingdom.

Renal Ischemia-Reperfusion Injury (IRI) is one of the main causes of Acute Kidney Injury (AKI), and may lead to chronic kidney disease. The high mortality rate of AKI has not changed in the last 5 decades due to non-recognition, nephrotoxin exposure, delayed diagnosis and lack of specific intervention. Complement activation plays important roles in IRI-induced AKI because of its association with immunity, inflammation, cell death and tissue repair. Read More

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http://dx.doi.org/10.2174/1566523218666180214093043DOI Listing
April 2019
3 Reads

How Do Dendritic Cells Play the Role in Ischemia/Reperfusion Triggered Kidney Allograft Rejection.

Curr Gene Ther 2017 ;17(6):400-404

Department of Advanced Technology for Transplantation, Osaka University Graduate School of Medicine, Osaka, Japan.

In deceased donors, Ischemia/Reperfusion Injury (IRI) is an important cause of allograft dysfunction. Prolonged cold and warm ischemia time leads to a high risk of early post-transplant complications, including acute and chronic rejection. Ischemia not only up-regulates inflammatory cytokines and chemokines, but also enhances the expression of MHC-class II and adhesion molecules on epithelial and dendritic cells. Read More

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http://dx.doi.org/10.2174/1566523218666180214095956DOI Listing
April 2019
17 Reads

Gene-modified Mesenchymal Stem Cell-based Therapy in Renal Ischemia- Reperfusion Injury.

Curr Gene Ther 2017 ;17(6):453-460

Department of Urology, The First Affiliated Hospital of Soochow University, 188 Shizi Rd, Suzhou 215006, China.

Acute Kidney Injury (AKI) is a common syndrome in the clinic and has become a worldwide public health problem. Renal Ischemia-Reperfusion Injury (IRI) is the most common cause of AKI. So far, effective treatment is still lacking for renal IRI, resulting in a high mortality rate of AKI. Read More

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http://dx.doi.org/10.2174/1566523218666180214094253DOI Listing
April 2019
4 Reads

Gene Therapy in Kidney Transplantation: Evidence of Efficacy and Future Directions.

Curr Gene Ther 2017 ;17(6):434-441

Department of Urology, Zhongshan Hospital, Fudan University, Shanghai, China.

Allograft loss remains a severe clinical problem after kidney transplantation. The molecular mechanism of graft loss is a complex process involving T and/or B cell activation, inflammation responses, autophagy and apoptosis. Since these pathways are involved in immune responses in kidney transplant rejection, application of genetic interference to inhibit specific pathways could present an effective targeted gene therapy method. Read More

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http://www.eurekaselect.com/159814/article
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http://dx.doi.org/10.2174/1566523218666180214095606DOI Listing
April 2019
8 Reads
2.540 Impact Factor

Epigenetic Regulation of Regulatory T Cells in Kidney Disease and Transplantation.

Curr Gene Ther 2017 ;17(6):461-468

Department of Respiration, Zhongshan Hospital, Fudan University, Shanghai 200032, China.

Regulatory T (Treg) cells are a kind of immunosuppression cells, which have been used to treat autoimmune diseases and induce allograft tolerance in clinical trials. While Treg cells based therapy is a promising treatment for kidney diseases and an emerging concept for tolerance induction in renal transplantation, a better understanding of the functions and biology of Treg cells is needed to be able to optimally exploit them. Epigenetics regulation, which refers to potentially heritable alterations in gene expression without underlying changes of the nucleotide sequence, plays an important role in Treg cells induction and maintenance. Read More

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http://dx.doi.org/10.2174/1566523218666180214093813DOI Listing
April 2019
6 Reads

CX3CL1/CX3CR1 Axis, as the Therapeutic Potential in Renal Diseases: Friend or Foe?

Curr Gene Ther 2017 ;17(6):442-452

Transplantation Center of the 3rd Xiangya Hospital, Central South University, Changsha, Hunan 410013, China.

The fractalkine receptor chemokine (C-X3-C motif) receptor 1 (CX3CR1) and its highly selective ligand CX3CL1 mediate chemotaxis and adhesion of immune cells, which are involved in the pathogenesis and progression of numerous inflammatory disorders and malignancies. The CX3CL1/CX3CR1 axis has recently drawn attention as a potential therapeutic target because it is involved in the ontogeny, homeostatic migration, or colonization of renal phagocytes. We performed a Medline/PubMed search to detect recently published studies that explored the relationship between the CX3CL1/CX3CR1 axis and renal diseases and disorders, including diabetic nephropathy, renal allograft rejection, infectious renal diseases, IgA nephropathy, fibrotic kidney disease, lupus nephritis and glomerulonephritis, acute kidney injury and renal carcinoma. Read More

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http://dx.doi.org/10.2174/1566523218666180214092536DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5902862PMC
April 2019
4 Reads