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Current Gene Therapy

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https://www.readbyqxmd.com/read/30058487/destination-penis-erectile-dysfunction-as-possible-future-indication-of-therapeutic-gene-delivery
#1
Serap Gur, Asim B Abdel-Mageed, Suresh C Sikka, Alma R Bartolome, Wayne J G Hellstrom
Erectile Dysfunction (ED) is a common health problem in roughly 50% of males of advanced age (40-70 years old). Recent attention related gene therapy to ED cases; this received much interest to further progress gene therapy ideals to ED treatment. This review is an attempt to analyze key challenges and to emphasize primary areas, including mostly preclinical and few clinical trials, cellular target(s), and different viral vectors/nanoparticles for gene delivery in ED. While overexpression of target genes can be silenced by RNA interference (RNAi), down-regulation of these mechanisms has been implicated in ED...
July 29, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29984652/phenotypic-characterization-of-a-family-with-an-in-frame-deletion-in-the-dmd-gene-and-variable-penetrance
#2
Inmaculada Perez-Sanchez, Maria Sabater-Molina, Maria Elisa Nicolas Rocamora, Guillermo Glover, Fuensanta Escudero, Pedro de Mingo Casado, Juan Ramon Gimeno-Blanes
Duchenne muscular dystrophy is a disorder with variable expression caused by frame-disrupting 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. Our aim was to study the clinical and genetic characteristics of a family with inherited cardiomyopathy and Becker muscular dystrophy. The index case was diagnosed with psychomotor retardation at 5 years of age...
July 9, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29303078/molecular-adjuvants-based-on-plasmids-encoding-protein-aggregation-domains-affect-bone-marrow-niche-homeostasis
#3
Maria Giovanna Sabbieti, Giovanna Lacava, Andrea Amaroli, Luigi Marchetti, Roberta Censi, Piera Di Martino, Dimitrios Agas
BACKGROUND: During last years, DNA vaccine immunogenicity has been optimized by the employment of co-stimulatory molecules and molecular adjuvants. It has been reported that plasmid (pATRex), encompassing the DNA sequence for the von Willebrand A (vWA/A) domain of the Anthrax Toxin Receptor-1 (ANTXR-1, alias TEM8, Tumor Endothelial Marker 8), acts as strong immune adjuvant by inducing formation of insoluble intracellular aggregates. Markedly, we faced with upsetting findings regarding the safety of pATRex as adjuvant since the aggregosome formation prompted to osteopenia in mice...
February 1, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29921205/current-advances-in-small-activating-rnas-for-gene-therapy-principles-applications-and-challenges
#4
Ling-Yan Zhou, Zhi-Yao He, Ting Xu, Yu-Quan Wei
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29874999/inhibition-of-west-nile-virus-replication-by-bifunctional-sirna-targeting-the-ns2a-and-ns5-conserved-region
#5
Divyanshi Karothia, Paban Kumar Dash, Manmohan Parida, Sameer Bhagyawant, Jyoti S Kumar
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. Two siRNAs were designed against the NS2A and NS5 regions of WNV which are highly conserved among Flaviviruses as it play important role in apoptosis and in viral replication respectively...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29769001/editorial-current-gene-therapy-on-non-viral-gene-delivery-and-therapy
#6
EDITORIAL
Lihua Peng
No abstract text is available yet for this article.
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29769000/preface-translational-gene-therapy-coming-of-age
#7
Lung-Ji Chang, Alfred S Lewin
No abstract text is available yet for this article.
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29749312/aav-mediated-overexpression-of-il-10-mitigates-the-inflammatory-cascade-in-stimulated-equine-chondrocyte-pellets
#8
Kyla F Ortved, Laila Begum, Darko Stefanovski, Alan J Nixon
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. Chondrocyte pellets were formed by centrifugation and were stimulated with IL-1β starting 48 hours following transduction...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29741137/editorial-genetics-and-gene-therapy-of-lysosomial-storage-disorders
#9
EDITORIAL
Antonino Tuttolomondo
No abstract text is available yet for this article.
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29714132/mir-7-replacement-therapy-in-parkinson-s-disease
#10
Ricardo Titze-de-Almeida, Simoneide Souza Titze-de-Almeida
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. Depletion of miR-7 in the substantia nigra of clinical samples is related to α-Syn accumulation, loss of dopaminergic cells, and reduction of dopamine in the striatum...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29651947/transplantation-of-bdnf-gene-recombinant-mesenchymal-stem-cells-and-adhesive-peptide-modified-hydrogel-scaffold-for-spinal-cord-repair
#11
Li-Ming Li, Ling-Ling Huang, Xin-Chi Jiang, Jia-Chen Chen, Hong-Wei OuYang, Jian-Qing Gao
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. However, transfection and in vivo gene expression of environmentally sensitive stem cells have been one of the most challenging subjects due to the requirement in both safety and transfection efficiency...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29637858/gene-therapy-to-enhance-bone-and-cartilage-repair-in-orthopaedic-surgery
#12
Sofia Bougioukli, Christopher H Evans, Ram K Alluri, Steven C Ghivizzani, Jay R Lieberman
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618310/genetics-and-gene-therapy-in-hunter-disease
#13
S Sestito, F Falvo, C Scozzafava, R Apa, L Pensabene, G Bonapace, M T Moricca, D Concolino
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618309/genetics-and-gene-therapy-of-anderson-fabry-disease
#14
Irene Simonetta, Antonino Tuttolomondo, Tiziana Di Chiara, Salvatore Miceli, Danai Vogiatzis, Francesca Corpora, Antonio Pinto
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618308/genetics-and-therapies-for-gm2-gangliosidosis
#15
Maria Begona Cachon-Gonzalez, Eva Zaccariotto, Timothy Martin Cox
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618307/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
#16
Haoyu Meng, Bo Chen, Zhengxian Tao, Zhihui Xu, Liansheng Wang, Ju Weizhu, Yongyong Hong, Xiang Liu, Hua Wang, Lisheng Wang, Zuze Wu, Zhijian Yang
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29512463/application-of-optogenetics-in-gene-therapy
#17
Toshihiro Kushibiki, Miya Ishihara
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29512462/dysfunction-in-brain-derived-neurotrophic-factor-signaling-pathway-and-susceptibility-to-schizophrenia-parkinson-s-and-alzheimer-s-diseases
#18
Alireza Mohammadi, Vahid Ghasem Amooeian, Ehsan Rashidi
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...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29493451/applications-and-prospects-of-non-viral-vectors-in-bone-regeneration
#19
Wei Yang, Fengzhe Wang, Longbao Feng, Shina Yan, Rui Guo
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. Cell activity stimulation, growth factors, and appropriate mechanical conditions are essential components of clinical treatment...
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29473500/crispr-cas-system-as-a-genome-engineering-platform-applications-in-biomedicine-and-biotechnology
#20
Atieh Hashemi
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...
2018: Current Gene Therapy
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