1,579 results match your criteria Journal of Gene Medicine [Journal]


Creating Cell and Animal Models of Human Disease by Genome Editing Using CRISPR/Cas9.

J Gene Med 2019 Feb 20:e3082. Epub 2019 Feb 20.

Department of Genetics, Shiraz University of Medical Science, Shiraz, Iran.

Aims: Set of unique sequences in bacterial genomes, responsible for protecting bacteria against bacteriophages, have been recently used for genetic manipulation of specific points in the genome. These system comprises of one RNA component and one enzyme component, known as CRISPR and Cas9, respectively. The present review focuses on the applications of the CRISPR/Cas9 technology in the development of cellular and animal models of human disease. Read More

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http://dx.doi.org/10.1002/jgm.3082DOI Listing
February 2019

Genetic polymorphism of Methylenetetrahydrofolate reductase is associated with insulin resistance in Egyptian women with polycystic ovary syndrome.

J Gene Med 2019 Feb 11:e3076. Epub 2019 Feb 11.

Clinical Pathology Department, Faculty of Medicine, Zagazig University, Egypt.

Introduction: A common polymorphism (677C to T; Ala to Val) in the methylenetetrahydrofolate reductase (MTHFR) gene is associated with decreased specific MTHFR activity and elevation of the homocysteine.

Aim: to investigate the association between single nucleotide polymorphism (SNP) in MTHFR 677C>T gene and insulin resistance in women with polycystic ovary syndrome (PCOS). Subject& Methods Two-hundred patients with PCOS were included in this case- control study. Read More

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http://dx.doi.org/10.1002/jgm.3076DOI Listing
February 2019
1 Read

Identification of microRNA expression in sentinel lymph nodes from patients with breast cancer via RNA sequencing for diagnostic accuracy.

J Gene Med 2019 Feb 4:e3075. Epub 2019 Feb 4.

Department of Ultrasonography, Peking University Shenzhen Hospital, Shenzhen, Guangdong, China.

Purpose: Sentinel lymph node (SLN) property assessment (with or without metastasis) is important while deciding the surgery for breast cancer; however, the current diagnosis of SLN metastasis remains to be studied. microRNAs (miRNAs) have been previously confirmed as a molecular marker for the diagnosis, development, and prognosis of tumors. However, the detailed role of miRNAs in the diagnosis of SLN metastasis has not been reported. Read More

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http://dx.doi.org/10.1002/jgm.3075DOI Listing
February 2019
1 Read
2.472 Impact Factor

LncRNA HCG11 suppresses the growth of glioma through cooperating with miR-4425/MTA3 axis.

J Gene Med 2019 Feb 1:e3074. Epub 2019 Feb 1.

Department of Neurosurgery, Shanghai Fourth People's Hospital, Shanghai, China.

Purpose: Glioma is a kind of malignant tumor which occurs in the central nervous system of adults. Long non-coding RNAs (lncRNAs) that potentially participate in the initiation and progression of glioma have been widely reported. As a now-found lncRNA, HLA complex group 11 (HCG11) has not been studied in glioma. Read More

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http://dx.doi.org/10.1002/jgm.3074DOI Listing
February 2019

Whole exome sequencing identified a novel mutation (p.Ala1884Pro) of β-spectrin in a Chinese family with hereditary spherocytosis.

J Gene Med 2019 Jan 28:e3073. Epub 2019 Jan 28.

Department of Cell Biology, The School of Life Sciences, Central South University, Changsha, China.

Background: Hereditary spherocytosis (HS) is an inherited disorder of erythrocyte. The typical feature of HS is the presence of spherical-shaped erythrocytes on the peripheral blood smear. According to previous studies, more than five candidate genes, such as ANK1, SPTB, SPTA1, SLC4A1 and EPB42 have been identified in HS patients. Read More

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http://dx.doi.org/10.1002/jgm.3073DOI Listing
January 2019
2 Reads

Ankyrin repeat domain 1: A novel gene for cardiac septal defects.

J Gene Med 2019 Jan 18:e3070. Epub 2019 Jan 18.

Department of Cardiac Surgery, Guangdong Cardiovascular Institute, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, Guangzhou, Guangdong, China.

Introduction: Cardiac septal defects account for more than 50% of congenital heart defects. Ankyrin repeat domain 1 (ANKRD1) is an important transcription factor that is mutated in multiple cardiac diseases; however, a relationship between the ANKRD1 mutation and cardiac septal defects has not been described.

Methods: We examined genetic mutations in a large family with three cardiac septal defect patients. Read More

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http://dx.doi.org/10.1002/jgm.3070DOI Listing
January 2019
1 Read

Association between the LIPG polymorphisms and serum lipid levels in the Maonan and Han populations.

J Gene Med 2019 Jan 18:e3071. Epub 2019 Jan 18.

Department of Cardiology, Institute of Cardiovascular Diseases, The First Affiliated Hospital, Guangxi Medical University, Nanning, Guangxi, China.

Introduction: The Maonan population is a relatively isolated minority in China. Little is known about endothelial lipase gene (LIPG) single nucleotide polymorphisms (SNPs) and serum lipid levels in the Chinese populations. The present study aimed to detect the association of several LIPG SNPs and environmental factors with serum lipid levels in the Chinese Maonan and Han populations. Read More

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http://doi.wiley.com/10.1002/jgm.3071
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January 2019
6 Reads

Adenoviral delivery of adiponectin ameliorates osteogenesis around implants in ovariectomized rats.

J Gene Med 2019 Jan 3:e3069. Epub 2019 Jan 3.

State Key Laboratory of Oral Diseases & National Clinical Research Center for Oral Diseases, Department of Oral and Maxillofacial Surgery, West China Hospital of Stomatology, Sichuan University, Chengdu, China.

Background: Adiponectin (APN) has been reported to promote bone formation. However, it is difficult to utilize a conventional method that administers sufficient APN to the implant site. The present study investigated the efficacy of an APN transgene to accelerate the implant osseointegration in ovariectomized (OVX) rats. Read More

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http://doi.wiley.com/10.1002/jgm.3069
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January 2019
2 Reads

Development of a liver-specific Tet-off AAV8 vector for improved safety of insulin gene therapy for diabetes.

J Gene Med 2019 Jan 20;21(1):e3067. Epub 2019 Jan 20.

Department of Medicine, National University of Singapore, Singapore.

Background: Diabetes mellitus is caused by a partial or complete lack of insulin production in the body. We have previously shown that a single injection of an adeno-associated virus serotype 8 (AAV8) vector carrying a modified and codon optimized human insulin gene induced hepatic production of insulin and corrected streptozotocin (STZ)-induced diabetes in mice for more than 1 year. Insulin production was constitutive, analogous to long-acting insulin therapy. Read More

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January 2019
1 Read

Corrigendum: Polymer-coated viral vectors: hybrid nanosystems for gene therapy.

Authors:

J Gene Med 2019 Jan;21(1):e3068

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http://dx.doi.org/10.1002/jgm.3068DOI Listing
January 2019
1 Read

P2RY12:rs7637803 TT variant genotype increases coronary artery aneurysm risk in Kawasaki disease in a southern Chinese population.

J Gene Med 2019 Jan 10;21(1):e3066. Epub 2019 Jan 10.

Department of Clinical Biological Resource Bank, Guangzhou Institute of Pediatrics, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, Guangdong, China.

Background: Activated-platelet increases the risk of thrombosis in Kawasaki disease (KD) patients with a coronary artery aneurysm (CAA). The ADP pathway is one of the platelet activation and aggregation pathways. The P2RY12 gene encodes the ADP receptor that is highly concentrated on platelets. Read More

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http://doi.wiley.com/10.1002/jgm.3066
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January 2019
4 Reads
2.472 Impact Factor

Long non-coding RNAs in cancer: Another layer of complexity.

J Gene Med 2019 Jan 8;21(1):e3065. Epub 2019 Jan 8.

Department of Genetics, Federal University of Parana, Curitiba, Brazil.

We review the most well characterized long non-coding RNAs (lncRNAs) with important roles in hallmarks of cancer, additionally including lncRNAs with a higher potential for clinical application. LncRNAs are transcripts larger than 200 nucleotides in length that do not appear to have protein-coding potential, although some of those may produce small functional peptides. These transcripts have attracted significant attention from researchers as a result of their role in genetic regulation, including epigenetic, transcriptional and post-transcriptional regulation, being involved in numerous biological processes, as well as being associated with multifactorial diseases, including tumorigenesis. Read More

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https://onlinelibrary.wiley.com/doi/abs/10.1002/jgm.3065
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January 2019
18 Reads

Cloning of carrier cells infected with oncolytic adenovirus driven by midkine promoter and biosafety studies.

J Gene Med 2018 Dec 13:e3064. Epub 2018 Dec 13.

Department of Obstetrics and Gynecology, School of Medicine, Ehime University, Ehime, Japan.

Background: A549 carrier cells infected with oncolytic adenovirus can induce complete tumor reduction of subcutaneous ovarian tumors but not intraperitoneal disseminated ovarian tumors. This appears to be a result of the insufficient antitumor effect of A549 carrier cells. Therefore, in the present study, we cloned a novel carrier cell with the aim of improving the antitumor effects. Read More

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http://dx.doi.org/10.1002/jgm.3064DOI Listing
December 2018
4 Reads

TP53 somatic mutations are associated with somatic mitogenome substitutions but not indels in colorectal cancer cells.

J Gene Med 2019 Jan 7;21(1):e3063. Epub 2019 Jan 7.

Division of Molecular and Forensic Genetics, Department of Forensic Medicine, Faculty of Medicine, Ludwik Rydygier Collegium Medicum, Nicolaus Copernicus University, Bydgoszcz, Poland.

Background: p53 is a tumour suppressor protein that is involved in many cancer-related processes. Growing evidence suggests that p53 also plays an important role in mitochondrial (mtDNA) maintenance. Somatic mitogenome mutations are frequently observed in colorectal cancer (CC) cells. Read More

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http://dx.doi.org/10.1002/jgm.3063DOI Listing
January 2019
1 Read

HIF-1α promotes bone marrow stromal cell migration to the injury site and enhances functional recovery after spinal cord injury in rats.

J Gene Med 2018 Dec 11;20(12):e3062. Epub 2018 Dec 11.

Department of Spine Surgery, Beijing Jishuitan Hospital, Beijing, China.

Background: Spinal cord injury (SCI) is a severe health problem worldwide, and efficacious strategies to properly repair SCI have not yet been developed. Recently, gene and cell therapies as alternative treatments for SCI have been proposed to comprise safe and promising strategies.

Methods: The present study investigated the therapeutic effects and underlying mechanisms of hypoxia-inducible factor-1α carried in recombinant adenovirus (Adv-HIF-1α), as administered immediately after SCI in adult rats. Read More

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http://dx.doi.org/10.1002/jgm.3062DOI Listing
December 2018
3 Reads

Telomere length, ZNF208 genetic variants and risk of chronic obstructive pulmonary disease in the Hainan Li population.

J Gene Med 2018 Dec 28;20(12):e3061. Epub 2018 Nov 28.

Department of General Practice, Hainan General Hospital, Haikou, Hainan, China.

Background: Chronic obstructive pulmonary disease (COPD) is a disease characterized by airflow limitation. It is not completely reversible and shows progressive development. ZNF208 rs8105767 affects telomere length, although the impact of telomere on COPD is still controversial. Read More

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http://doi.wiley.com/10.1002/jgm.3061
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December 2018
5 Reads

Enhancing atrial-specific gene expression using a calsequestrin cis-regulatory module 4 with a sarcolipin promoter.

J Gene Med 2018 Dec 4;20(12):e3060. Epub 2018 Dec 4.

Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

Background: Cardiac gene therapy using the adeno-associated virus serotype 9 vector is widely used because of its efficient transduction. However, the promoters used to drive expression often cause off-target localization. To overcome this, studies have applied cardiac-specific promoters, although expression is debilitated compared to that of ubiquitous promoters. Read More

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http://dx.doi.org/10.1002/jgm.3060DOI Listing
December 2018
10 Reads
2.472 Impact Factor

Discordance in TLR2 (-196 to -174) polymorphism effect on HIV infection risk.

J Gene Med 2018 Oct 12;20(10-11):e3051. Epub 2018 Oct 12.

Surgery, Biochemistry and Immunology Department, School of Medicine, University of Malaga, Málaga, Spain.

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http://doi.wiley.com/10.1002/jgm.3051
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http://dx.doi.org/10.1002/jgm.3051DOI Listing
October 2018
9 Reads

IL1R1 and IL1R2 polymorphisms were associated with tuberculosis risk: A pilot study.

J Gene Med 2018 Oct 18;20(10-11):e3057. Epub 2018 Oct 18.

Department of Respiratory and critical diseases medicine, the First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi, China.

Background: Interleukin (IL)-1 has been reported to be involved in the development of tuberculosis (TB). IL1R1 and IL1R2 encode a cytokine receptor that belongs to the IL-1 receptor family. However, few studies have reported on the polymorphisms of IL1R1 and IL1R2 in TB patients. Read More

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http://doi.wiley.com/10.1002/jgm.3057
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http://dx.doi.org/10.1002/jgm.3057DOI Listing
October 2018
12 Reads

Targeting dipeptidyl peptidase 8 genes inhibits proliferation, migration and invasion by inhibition of cyclin D1 and MMP2MMP9 signal pathway in cervical cancer.

J Gene Med 2018 Dec 8;20(12):e3056. Epub 2018 Nov 8.

Department of Obstetrics and Gynecology, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, China.

Background: DPP8 is a member of the dipeptidyl peptidase IV family, which belongs to the S9b protease subfamily. It regulates cell proliferation, apoptosis, migration and invasion during cancer progression.

Methods: To investigate the role of DPP8 in cervical cancer, we examined DPP8 levels in cervical cancer tissues and cells. Read More

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http://dx.doi.org/10.1002/jgm.3056DOI Listing
December 2018
2 Reads
2.470 Impact Factor

Association between PTCH1 and RAD54B single-nucleotide polymorphisms and non-syndromic orofacial clefts in a northern Chinese population.

J Gene Med 2018 Dec 5;20(12):e3055. Epub 2018 Nov 5.

Department of Oral and Maxillofacial Surgery, the First Affiliated Hospital of Harbin Medical University, Heilongjiang, China.

Background: Non-syndromic orofacial clefts (NSOC) is one of the most common congenital malformations, and its etiology involves both genetic and environmental factors. The present aimed to investigate the association of six single nucleotide polymorphisms (SNPs) (rs10512248 in PTCH1, rs12681366 and rs958447 in RAD54B, rs13317 in FGFR1, rs1838105 and rs4968247 in WNT9B) with NSOC in a Northern Chinese population.

Methods: In the present study, HI-SNP technology was used to conduct genotyping of the six SNPs (rs10512248, rs12681366, rs957448, rs13317, rs1838105 and rs4968247) in 596 patients with NSOC and 466 healthy individuals from a Northern Chinese population. Read More

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http://dx.doi.org/10.1002/jgm.3055DOI Listing
December 2018
2 Reads

Arg399Gln substitution in XRCC1 as a prognostic and predictive biomarker for prostate cancer: Evidence from 8662 subjects and a structural analysis.

J Gene Med 2018 Oct 26;20(10-11):e3053. Epub 2018 Sep 26.

Gametogenesis Research Center, Kashan University of Medical Sciences, Kashan, Iran.

Background: The Arg399Gln polymorphism in the X-ray repair cross-complementing group 1 gene (XRCC1) may alter the risk of prostate cancer (PCa). The present study aimed to investigate the association of the XRCC1-Arg399Gln polymorphism with PCa risk in an Iranian population, as followed by a meta-analysis and an in silico analysis.

Methods: In a case-control study, 360 subjects were included (180 men with PCa and 180 healthy controls). Read More

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http://dx.doi.org/10.1002/jgm.3053DOI Listing
October 2018
15 Reads

The association between polymorphism of the BDNF gene and cigarette smoking in the Iranian population.

J Gene Med 2018 Oct 12;20(10-11):e3052. Epub 2018 Sep 12.

Department of Medical Genetics and Molecular Biology, Faculty of Medicine, Iran University of Medical Sciences, Tehran, Iran.

Background: Cigarette smoking remains a serious public health problem. Environmental and genetic factors both play critical roles in the process of cigarette smoking. We aimed to investigate the associations between single nucleotide polymorphisms (SNP) in the BDNF gene and cigarette smoking in the Iranian population. Read More

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http://dx.doi.org/10.1002/jgm.3052DOI Listing
October 2018
5 Reads

Minimal conditioning in Fanconi anemia promotes multi-lineage marrow engraftment at 10-fold lower cell doses.

J Gene Med 2018 Oct 1;20(10-11):e3050. Epub 2018 Oct 1.

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.

Background: Gene therapy approaches for the treatment of Fanconi anemia (FA) hold promise for patients without a suitably matched donor for an allogeneic bone marrow transplant. However, significant limitations include the collection of sufficient stem cell numbers from patients, the fragility of these cells during ex vivo manipulation, and clinically meaningful engraftment following transplantation. With these challenges in mind, we were interested in determining (i) whether gene-corrected cells at progressively lower numbers can successfully engraft in FA; (ii) whether low-dose conditioning facilitates this engraftment; and (iii) whether these cells can be selected for post-transplant. Read More

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http://doi.wiley.com/10.1002/jgm.3050
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October 2018
14 Reads

Polymorphisms in TH1-TH2 cytokine and receptor genes associated with risk of vertical HIV transmission, in Mumbai, India.

J Gene Med 2018 Oct 4;20(10-11):e3047. Epub 2018 Sep 4.

Department of Infectious Diseases Biology, National Institute for Research in Reproductive Health (DHR/ICMR), Mumbai, Maharashtra, India.

Background: Vertical HIV transmission does not occur in all exposed infants. Many infants remain HIV uninfected even after exposure. This is partly attributed to the host genes involving cytokine production, which is rarely documented in vertical transmission. Read More

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http://dx.doi.org/10.1002/jgm.3047DOI Listing
October 2018
22 Reads

Neuraminidase-mediated desialylation augments AAV9-mediated gene expression in skeletal muscle.

J Gene Med 2018 Sep 4;20(9):e3049. Epub 2018 Sep 4.

Department of Medicine, Division of Cardiovascular Medicine, University of Virginia, Charlottesville, VA, USA.

Background: Following systemic delivery, AAV9-mediated gene expression is significantly increased in ischemic versus non-ischemic muscle, suggesting that AAV9 is an attractive vector for treating peripheral arterial disease. Potential mechanisms underlying ischemia-augmented expression include: (i) increased vascular permeability and (ii) "unmasking" of endogenous AAV9 receptors. In the present study, we aimed to reconstitute the ischemic induction of AAV9 in vivo, using local injection of histamine (to increase vascular permeability) and neuraminidase (to desialylate cell surface glycans). Read More

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http://doi.wiley.com/10.1002/jgm.3049
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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6209320PMC
September 2018
44 Reads

Association of genetic polymorphisms in VEGF -460, -7 and -583 and hematocrit level with the development of idiopathic recurrent pregnancy loss and a meta-analysis.

J Gene Med 2018 Sep 30;20(9):e3048. Epub 2018 Aug 30.

Department of Biomedical Science, College of Life Science, CHA University, Seongnam, South Korea.

Background: The present study was performed to investigate whether genetic variants of VEGF are associated with recurrent pregnancy loss (RPL) in Korean women and to provide insight into the role of VEGF in the pathogenesis of RPL development.

Methods: A cohort of 384 women with idiopathic RPL with a history of two or more uxexplained consecutive early pregnancy losses and 236 control women were recruited from an infertility center of university-teaching hospital in Korea between March 1999 and February 2010. We examined three VEGF polymorphisms (rs833061, rs3025020 and rs25648). Read More

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http://dx.doi.org/10.1002/jgm.3048DOI Listing
September 2018
4 Reads

Transduction efficacy and retrograde movement of a lentiviral vector pseudotyped by modified rabies glycoprotein throughout the trisynaptic circuit of the rat hippocampus.

J Gene Med 2018 Sep 15;20(9):e3046. Epub 2018 Aug 15.

Department of Physiology and Pharmacology, Pasteur Institute of Iran, Tehran, Iran.

Background: The trisynaptic circuit (entorhinal cortex-dentate gyrus-CA3-CA1) is a key unidirectional network in the hippocampus. Damage to the hippocampus interrupts this circuit and causes neurological disorders. Efficient delivery of therapeutic genes into this network is of great interest with respect to treating trisynaptic circuit pathologies. Read More

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http://dx.doi.org/10.1002/jgm.3046DOI Listing
September 2018
3 Reads

MIF-173G/C (rs755622) polymorphism as a risk factor for acute lymphoblastic leukemia development in children.

J Gene Med 2018 Sep 2;20(9):e3044. Epub 2018 Aug 2.

Department of Biochemistry, Faculty of Sciences, Damietta University, New Damietta, Egypt.

Background: Macrophage inhibitory factor (MIF) is a pro-inflammatory cytokine modulating monocyte motility and a pleiotropic regulator of different biological and cellular processes. The MIF-173G/C (rs755622) polymorphism is found in the promoter region and affects its activity. The present study investigated the MIF polymorphism as a risk factor for the development of acute lymphoblastic leukemia (ALL) in Egyptian children. Read More

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http://dx.doi.org/10.1002/jgm.3044DOI Listing
September 2018
5 Reads

Thymosin β4 suppresses CCl -induced murine hepatic fibrosis by down-regulating transforming growth factor β receptor-II.

J Gene Med 2018 Sep 15;20(9):e3043. Epub 2018 Aug 15.

Department of Rheumatology, The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi Province, China.

Background: The present study aimed to clarify the effects of thymosin β4 (Tβ4) on CCl -induced hepatic fibrosis in mice and to further explore the underlying mechanisms.

Methods: Expression of Tβ4 in fibrotic liver tissues was assessed by a quantitative real time-reverse transcriptase polymerase chain reaction and immunohistochemistry. The effects of intraperitoneal adeno-associated virus-Tβ4 (AAV-Tβ4) on CCl -induced hepatic fibrosis were observed by the evaluation of collagen deposition, hepatic stellate cell (HSC) activation and pro-fibrotic cytokine expression. Read More

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http://dx.doi.org/10.1002/jgm.3043DOI Listing
September 2018
7 Reads

Bone morphogenetic protein-2 is a stronger inducer of osteogenesis within muscle tissue than heterodimeric bone morphogenetic protein-2/6 and -2/7: Implications for expedited gene-enhanced bone repair.

J Gene Med 2018 Sep 24;20(9):e3042. Epub 2018 Jul 24.

Department of Orthopedic Surgery, Physical Medicine and Rehabilitation, University Hospital Grosshadern, Ludwig-Maximilians-University Munich, Munich, Germany.

Background: Bone morphogenetic protein (BMP)-2 gene-activated muscle tissue fragments can regenerate large bone defects in preclinical animal models. The use of tissue fragments instead of isolated cells expedites gene-enhanced tissue engineering and may increase the possibility of clinical translation. The present in vitro study investigated whether the osteoinductive effect of BMP-2 on muscle tissue fragments can be enhanced using the heterodimers BMP-2/6 or BMP-2/7. Read More

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http://dx.doi.org/10.1002/jgm.3042DOI Listing
September 2018
11 Reads

Epidermal growth factor receptor targeted methotrexate and small interfering RNA co-delivery.

J Gene Med 2018 Jul 17;20(7-8):e3041. Epub 2018 Jul 17.

Pharmaceutical Biotechnology, Department of Pharmacy, Ludwig-Maximilians-Universität München, Munich, Germany.

Background: Developing new drug delivery carriers addressing chemoresistance is still full of challenges and opportunities. As the rapid development of small interfering RNA (siRNA) provides promising therapeutic perspectives, nanocarriers for drug and siRNA co-delivery present new alternatives for cancer therapy.

Methods: A co-delivery nanosystem for methotrexate (MTX) or gamma-glutamylated derivatives (gE -MTX and gE -MTX) and antitumoral EG5 siRNA has been developed utilizing the sequence defined cationic lipo-oligomers 454, 1021 and 1027. Read More

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http://dx.doi.org/10.1002/jgm.3041DOI Listing
July 2018
18 Reads

Efficacy and safety of a clinically relevant foamy vector design in human hematopoietic repopulating cells.

J Gene Med 2018 Jul 19;20(7-8):e3028. Epub 2018 Jul 19.

Department of Pharmaceutical Sciences, Washington State University College of Pharmacy, WSU Spokane, Spokane, WA, USA.

Background: Previous studies have shown that foamy viral (FV) vectors are a promising alternative to gammaretroviral and lentiviral vectors and also that insulators can improve FV vector safety. However, in a previous analysis of insulator effects on FV vector safety, strong viral promoters were used to elicit genotoxic events. In the present study, we developed and analyzed the efficacy and safety of a high-titer, clinically relevant FV vector driven by the housekeeping promoter elongation factor-1α and insulated with an enhancer blocking A1 insulator (FV-EGW-A1). Read More

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http://dx.doi.org/10.1002/jgm.3028DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6368973PMC
July 2018
2 Reads

Comparative analysis and optimization of protocols for producing recombinant lentivirus carrying the anti-Her2 chimeric antigen receptor gene.

J Gene Med 2018 Jul 6;20(7-8):e3027. Epub 2018 Jul 6.

Key Laboratory of Laboratory Medicine, Ministry of Education, Zhejiang Provincial Key Laboratory of Medical Genetics, School of Laboratory Medicine and Life Sciences, Wenzhou Medical University, Wenzhou, Zhejiang, China.

Background: The production of anti-Her2 chimeric antigen receptor (CAR) T cells needs to be optimized to make it a reliable therapy.

Methods: Three types of lentiviral vectors expressing anti-Her2 CAR together with packaging plasmids were co-transfected into 293 T-17 cells. The vector with the best packaging efficiency was selected, and the packaging cell culture system and packaging plasmid system were optimized. Read More

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http://doi.wiley.com/10.1002/jgm.3027
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July 2018
19 Reads

Human interleukin-10 delivered intrathecally by self-complementary adeno-associated virus 8 induces xenogeneic transgene immunity without clinical neurotoxicity in swine.

J Gene Med 2018 Jul 15;20(7-8):e3026. Epub 2018 Jun 15.

Departments of Anesthesiology and Oncology, Mayo Clinic, Translational Science Track, Mayo Graduate School, Rochester, MN, USA.

Introduction: Intrathecal interleukin (IL)-10 delivered by plasmid or viral gene vectors has been proposed for clinical testing because it is effective for chronic pain in rodents, is a potential therapeutic for various human diseases, and was found to be nontoxic in dogs, when the human IL-10 ortholog was tested. However, recent studies in swine testing porcine IL-10 demonstrated fatal neurotoxicity. The present study aimed to deliver vector-encoded human IL-10 in swine, measure expression of the transgene in cerebrospinal fluid and monitor animals for signs of neurotoxicity. Read More

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http://dx.doi.org/10.1002/jgm.3026DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6105440PMC
July 2018
2 Reads

Palmitoylated phosphodiester gapmer designs with albumin binding capacity and maintained in vitro gene silencing activity.

J Gene Med 2018 Jul 25;20(7-8):e3025. Epub 2018 Jun 25.

The Interdisciplinary Nanoscience Center (iNANO), Department of Molecular Biology and Genetics, Aarhus University, Aarhus C, Denmark.

Background: Antisense gapmer oligonucleotide drugs require delivery and biodistribution enabling technologies to increase in vivo efficacy. An attractive approach is their binding and consequent transport by the endogenous human serum albumin pool as mediated by fatty acid incorporation into the gapmer design.

Methods: The present study investigated the effect of palmitoyl modification and position on albumin-binding, cellular uptake and in vitro gene silencing of gapmers with either a phosphorothioate (PS) or phosphodiester (PO) backbone. Read More

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http://dx.doi.org/10.1002/jgm.3025DOI Listing
July 2018
4 Reads

Soluble Fgl2 restricts autoimmune hepatitis progression via suppressing Tc17 and conventional CD8+ T cell function.

J Gene Med 2018 Jul 19;20(7-8):e3023. Epub 2018 Jun 19.

Institute of Infectious Disease, Tongji Hospital of Tongji Medical College, Wuhan, China.

Background: Autoimmune hepatitis (AIH) is an inflammatory disease caused by an aberrant immune response to hepatic self-antigens in which regulatory T cells (Tregs) are critical for maintaining immunosupression. The soluble form of fibrinogen-like protein 2 (sFGL2), a novel effector molecule of Treg, is rarely investigated in AIH. In the present study, we dissected the role of sFGL2 in autoimmune hepatitis and its potential mechanism underlying AIH progression. Read More

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http://dx.doi.org/10.1002/jgm.3023DOI Listing
July 2018
3 Reads

The VEGFA -1154G/A polymorphism is associated with reduced risk of rheumatoid arthritis but not with systemic lupus erythematosus in Mexican women.

J Gene Med 2018 Jun 5;20(6):e3024. Epub 2018 Jun 5.

Laboratorio de Genómica del Cáncer, Instituto Nacional de Medicina Genómica, Mexico City, Mexico.

Background: Levels of circulating vascular endothelial growth factor (VEGF) (a potent endothelial-cell-specific angiogenic factor) have been correlated with disease activity in patients with rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE). In addition, several single nucleotide polymorphisms (SNPs), including the VEGFA -2578C/A, have been associated with RA in some populations. By contrast, the role of different VEGFA SNPs in the susceptibility to SLE has received little attention. Read More

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http://dx.doi.org/10.1002/jgm.3024DOI Listing
June 2018
5 Reads

MiR-21 ameliorates age-associated skin wound healing defects in mice.

J Gene Med 2018 Jun 3;20(6):e3022. Epub 2018 May 3.

Institute of Combined Injury, State Key Laboratory of Trauma, Burn and Combined Injury, Chongqing Engineering Research Center for Nanomedicine, College of Preventive Medicine, Army Military Medical University, Chongqing, China.

Background: The cellular and molecular mechanisms responsible for the age-associated delay of cutaneous wound healing are still not well understood. Previous studies have shown that miR-21 plays key roles during skin wound healing. We presumed that dysregulation of miR-21 may be involved in age-associated defects in wound healing and that miR-21 may be one potential therapeutic target by which to ameliorate wound defects in elderly subjects. Read More

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http://doi.wiley.com/10.1002/jgm.3022
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http://dx.doi.org/10.1002/jgm.3022DOI Listing
June 2018
30 Reads

Gene therapy for Glut1-deficient mouse using an adeno-associated virus vector with the human intrinsic GLUT1 promoter.

J Gene Med 2018 Apr 6;20(4):e3013. Epub 2018 Apr 6.

Department of Pediatrics, Jichi Medical University, Tochigi, Japan.

Background: We generated an adeno-associated virus (AAV) vector in which the human SLC2A1 gene, encoding glucose transporter type 1 (GLUT1), was expressed under the human endogenous GLUT1 promoter (AAV-GLUT1). We examined whether AAV-GLUT1 administration could lead to functional improvement in GLUT1-deficient mice.

Methods: We extrapolated human endogenous GLUT1 promoter sequences from rat minimal Glut1 promoter sequences. Read More

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http://doi.wiley.com/10.1002/jgm.3013
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http://dx.doi.org/10.1002/jgm.3013DOI Listing
April 2018
43 Reads

Designer nuclease-mediated gene correction via homology-directed repair in an in vitro model of canine hemophilia B.

J Gene Med 2018 May 3;20(5):e3020. Epub 2018 May 3.

Institute of Virology and Microbiology, Center for Biomedical Education and Research (ZBAF), Department of Human Medicine, Faculty of Health, Witten/Herdecke University, Witten, Germany.

Background: Gene correction at specific target loci provides a powerful strategy for overcoming genetic diseases. In the present study, we aimed to use an in vitro model for canine hemophilia B containing a single point mutation in the catalytic domain of the canine coagulation factor IX (cFIX) gene. To correct the defective gene via homology-directed repair (HDR), we designed transcription-activator like effector nucleases and clustered regularly interspaced short palindromic repeats including Cas9 (CRISPR/Cas9) for introduction of double-strand breaks at the mutation site. Read More

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http://dx.doi.org/10.1002/jgm.3020DOI Listing
May 2018
5 Reads

Cytokine-induced interleukin-1 receptor antagonist protein expression in genetically engineered equine mesenchymal stem cells for osteoarthritis treatment.

J Gene Med 2018 May 22;20(5):e3021. Epub 2018 Apr 22.

Institute of Pathology and Forensic Veterinary Medicine, Working Group Histology and Embryology, University of Veterinary Medicine Vienna, Vienna, Austria.

Background: A combination of tissue engineering methods employing mesenchymal stem cells (MSCs) together with gene transfer takes advantage of innovative strategies and highlights a new approach for targeting osteoarthritis (OA) and other cartilage defects. Furthermore, the development of systems allowing tunable transgene expression as regulated by natural disease-induced substances is highly desirable.

Methods: Bone marrow-derived equine MSCs were transduced with a lentiviral vector expressing interleukin-1 receptor antagonist (IL-1Ra) gene under the control of an inducible nuclear factor-kappa B-responsive promoter and IL-1Ra production upon pro-inflammatory cytokine stimulation [tumor necrosis factor (TNF)α, interleukin (IL)-1β] was analysed. Read More

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http://dx.doi.org/10.1002/jgm.3021DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6001542PMC
May 2018
15 Reads

A clinical guidance to DFNA22 drawn from a Korean cohort study with an autosomal dominant deaf population: A retrospective cohort study.

J Gene Med 2018 Jun 30;20(6):e3019. Epub 2018 Apr 30.

Department of Otorhinolaryngology, Seoul National University Bundang Hospital, Seongnam, Korea.

Background: The MYO6 gene, if altered, can cause nonsyndromic hearing loss (NSHL) either in an autosomal dominant (AD) (DFNA22) or recessive form. The present study identified MYO6 variants in the cohort of Korean AD NSHL families and investigated the audiological phenotypes of DFNA22 with respect to suggesting clinical guides for the counseling of DFNA22.

Methods: A retrospective cohort study was performed on 81 AD NSHL families in two hospitals. Read More

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http://dx.doi.org/10.1002/jgm.3019DOI Listing
June 2018
6 Reads
2.470 Impact Factor

Recent advances in gene-enhanced bone tissue engineering.

J Gene Med 2018 Jun 9;20(6):e3018. Epub 2018 May 9.

Center for Rehabilitation, RKU - University and Rehabilitation Hospitals Ulm, Ulm, Germany.

The loss of bone tissue represents a critical clinical condition that is frequently faced by surgeons. Substantial progress has been made in the area of bone research, providing insight into the biology of bone under physiological and pathological conditions, as well as tools for the stimulation of bone regeneration. The present review discusses recent advances in the field of gene-enhanced bone tissue engineering. Read More

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http://dx.doi.org/10.1002/jgm.3018DOI Listing
June 2018
3 Reads

MicroRNA-guided gene expression in prostate cancer: Literature and database overview.

J Gene Med 2018 May 30;20(5):e3016. Epub 2018 Apr 30.

Institute of Chemical Biology and Fundamental Medicine, Siberian Branch, Russian Academy of Sciences, Novosibirsk, Russia and 'E. Meshalkin National Medical Research Center' of the Ministry of Health of the Russian Federation, Novosibirsk, Russia.

Insight into the aberrant expression of microRNAs (miRNAs) and the genes that they regulate during the progression of cancer in general and prostate cancer (PCa) in particular is one of the most important issues in current molecular biomedicine and allows for the discovery of therapeutic or diagnostic miRNA targets. The present study aimed to analyze the available data regarding the direct or indirect effects of miRNAs on the expression of the mRNAs involved in carcinogenesis and to enable updating and optimizing the selection of the corresponding targets. The present review focuses on the data related to the genes with miRNA-dependent expression during the development of PCa. Read More

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http://dx.doi.org/10.1002/jgm.3016DOI Listing
May 2018
3 Reads

Partial pancreatic transdifferentiation of primary human hepatocytes in the livers of a humanised mouse model.

J Gene Med 2018 May 16;20(5):e3017. Epub 2018 Apr 16.

School of Life Sciences, University of Technology Sydney, Sydney, Australia.

Background: Gene therapy is one treatment that may ultimately cure type 1 diabetes. We have previously shown that the introduction of furin-cleavable human insulin (INS-FUR) to the livers in several animal models of diabetes resulted in the reversal of diabetes and partial pancreatic transdifferentiation of liver cells. The present study investigated whether streptozotocin-diabetes could be reversed in FRG mice in which chimeric mouse-human livers can readily be established and, in addition, whether pancreatic transdifferentiation occurred in the engrafted human hepatocytes. Read More

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http://dx.doi.org/10.1002/jgm.3017DOI Listing
May 2018
7 Reads

Gene therapy clinical trials worldwide to 2017: An update.

J Gene Med 2018 May 19;20(5):e3015. Epub 2018 Apr 19.

Department of Laboratory Medicine, Uppsala University Hospital, Uppsala, Sweden.

To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. Read More

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http://doi.wiley.com/10.1002/jgm.3015
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http://dx.doi.org/10.1002/jgm.3015DOI Listing
May 2018
68 Reads

Analysis and dissociation of anti-HIV effects of shRNA to CCR5 and the fusion inhibitor C46.

J Gene Med 2018 Feb;20(2-3):e3006

Faculty of Medicine, UNSW Australia, Sydney, NSW, Australia.

Background: The gene therapeutic Cal-1 comprises the anti-HIV agents: (i) sh5, a short hairpin RNA to CCR5 that down-regulates CCR5 expression and (ii) maC46 (C46), a peptide that inhibits viral fusion with the cell membrane. These constructs were assessed for inhibition of viral replication and selective cell expansion in a number of settings.

Methods: HIV replication, selective outgrowth and cell surface viral binding were analysed with a single cycle infection assay of six pseudotyped HIV strains and a static and longitudinal passaging of MOLT4/CCR5 cells with HIV. Read More

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http://dx.doi.org/10.1002/jgm.3006DOI Listing
February 2018
6 Reads

MicroRNA expression data analysis to identify key miRNAs associated with Alzheimer's disease.

J Gene Med 2018 Jun 21;20(6):e3014. Epub 2018 May 21.

Shanghai Tenth People's Hospital, School of Medicine, Tongji University, Shanghai, China.

Background: MicroRNAs (miRNAs) have become increasingly prevalent as a result of the association of their deregulation with neurodegenerative disorders, especially Alzheimer's disease (AD). However, the association between miRNAs and AD remains unclear.

Methods: In the present study, Nine representative miRNA datasets were selected for the identification of the critical miRNAs by analyzing the overlapping relationships among them. Read More

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http://dx.doi.org/10.1002/jgm.3014DOI Listing
June 2018
8 Reads