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Current Gene Therapy

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https://www.readbyqxmd.com/read/29651947/transplantation-of-bdnf-gene-recombinant-mesenchymal-stem-cells-and-adhesive-peptide-modified-hydrogel-scaffold-for-spinal-cord-repair
#1
Li-Ming Li, Ling-Ling Huang, Xin-Chi Jiang, Jia-Chen Chen, Hong-Wei OuYang, Jian-Qing Gao
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...
April 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29637858/gene-therapy-to-enhance-bone-and-cartilage-repair-in-orthopaedic-surgery
#2
Sofia Bougioukli, Christopher Evans, Ram Alluri, Steve Ghivizzani, Jay 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...
April 10, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618310/genetics-and-gene-therapy-in-hunter-disease
#3
Sestito Simona, Francesca Falvo, Rosalbina Apa, Licia Pensabene, Giuseppe Bonapace, Maria Teresa Moricca, Daniela Concolino
Mucopolysaccharidosis type II or Hunter syndrome is a rare X-linked lysosomal storage disorder due to a mutation in the gene encoding the lysosomal enzyme iduronate-2-sulfatase. The consequent enzyme deficiency leads to a progressive, multisystem accumulation of glycosaminoglycans throughout the body, 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 current available therapies for Hunter syndrome, hematopoietic stem cell transplantation and recombinant enzyme replacement therapy, mainly regarding brain achievement, encouraged several studies which recognized gene therapy as a potential therapeutic option for this condition...
April 4, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618309/genetics-and-gene-therapy-of-anderson-fabry-disease
#4
Irene Simonetta, Antonino Tuttolomondo, Tiziana Di Chiara, Salvatore Miceli
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 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 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...
April 4, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29618308/genetics-and-therapies-for-gm2-gangliosidosis
#5
María 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...
April 4, 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
#6
Haoyu Meng, Bo Chen, Zhengxian Tao, Zhihui Xu, Liansheng Wang, Ju Weizhu, Yongyong Hong, Xiang Liu, Hua Wang, Lisheng Wang, Zuze Wu, Zhijian Yan
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 evaluated 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 post-infarct heart failure who were not indicated to revascularization and had received the optimal standardized medication therapy were included in the study...
April 4, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29512463/application-of-optogenetics-in-gene-therapy
#7
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...
March 2, 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
#8
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...
March 2, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29493451/applications-and-prospects-of-non-viral-vectors-in-bone-regeneration
#9
Wei Yang, Fengzhe Wang, Longbao Feng, Shina Yan, Rui Guo
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. However, growth factors tend to degrade over time in the human body...
February 27, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29473500/crispr-cas-system-as-a-genome-engineering-platform-applications-in-biomedicine-and-biotechnology
#10
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 tools 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. First, this review aims to describe the biology of CRISPR system...
February 20, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446741/what%C3%A2-s-new-in-gene-therapy-of-hemophilia
#11
E Carlos Rodriguez-Merchan
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. RESULTS: The most promising vectors for hemophilia GT are adeno-associated virus (AAV) and lentivirus...
February 14, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446738/how-do-dendritic-cells-play-the-role-in-ischemia-reperfusion-triggered-kidney-allograft-rejection
#12
Songjie Cai, Naotsugu Ichimaru, Shiro Takahara
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. Moreover, the danger associated molecular patterns (DAMPs) released from stressed or dying cells, not only cause or amplify tissue inflammation and trigger tissue repair in response to IRI, but also act as adjuvants that enhance DC maturation and potentiate the adaptive immune response...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446737/gene-modified-mesenchymal-stem-cell-based-therapy-in-renal-ischemia-reperfusion-injury
#13
Hongbo Xu, Cheng Chen, Linkun Hu, Jianquan Hou
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. Mesenchymal stem cells (MSCs), considered as a promising candidate for tissue repair and regenerative medicine have aroused an increasing concern in recent years for the capacity of self-renewal and multi-lineage differentiation...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29366415/dosage-and-passage-dependent-neuroprotective-effects-of-exosomes-derived-from-rat-bone-marrow-mesenchymal-stem-cells-an-in-vitro-analysis
#14
Chaitra Venugopal, Christopher Shamir, Sivapriya Senthilkumar, Janitri Venkatachala Babu, Peedikayil Kurien Sonu, Kusum Jain Nishtha, Kiranmai S Rai, K Shobha, Anandh Dhanushkodi
BACKGROUND: Neurodegenerative diseases comprise a group of disorders for which no treatment is available till date. Stem cell based therapy offers great hope and promise. However, stem cell transplantation is associated with certain disadvantages like poor targeted migration, engraftment and survival of the transplanted cells. MATERIAL & METHOD: Exosomes, a type of extracellular membrane vesicle released by all cell types including stem cells, offer an alternative to stem cell transplantation...
January 24, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29303078/molecular-adjuvants-based-on-plasmids-encoding-protein-aggregation-domains-affect-bone-marrow-niche-homeostasis
#15
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/29357792/nonviral-delivery-systems-for-cancer-gene-therapy-strategies-and-challenges
#16
Gayong Shim, Dongyoon Kim, Quoc-Viet Le, Gyu Thae Park, Taekhyun Kwon, Yu-Kyoung Oh
Gene therapy has been receiving widespread attention due to its unique advantage in regulating the expression of specific target genes. In the field of cancer gene therapy, modulation of gene expression has been shown to decrease oncogenic factors in cancer cells or increase immune responses against cancer. Due to the macromolecular size and highly negative physicochemical features of plasmid DNA, efficient delivery systems are an essential ingredient for successful gene therapy. To date, a variety of nanostructures and materials have been studied as nonviral gene delivery systems...
January 19, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29357791/neurodegenerative-disorders-treatment-the-microrna-role
#17
Barbara Ridolfi, Hanin Abdel-Haq
Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease and prion disease are not timely and effectively treated using conventional therapies. This emphasizes the need for alternative therapeutic approaches. In this respect, gene-based therapies have been adopted as potentially feasible alternative therapies, where the microRNA (miRNA) approach has experienced a great explosion in recent years. Because miRNAs have been shown to be implicated in the pathogenesis of several diseases including neurodegenerative diseases, they are intensely studied as candidates for diagnostic and prognostic biomarkers, as predictors of drug response and as therapeutic agents...
January 19, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/28595562/non-viral-delivery-systems-for-breast-cancer-gene-therapy
#18
Golnaz Vaseghi, Laleh Rafiee, Shaghayegh Haghjooy Javanmard
INTRODUCTION: The ever-evolving field of gene therapy promises several innovative treatments for cancer. Advances in genetic modification of tumor cells and micro environment have led to the development of more effective therapeutic strategies with fewer side effects. MATERIAL & METHODS: The development of effective delivery system challenges, remains. Non-viral vectors are interesting due to their bio-safety and their ability to transfer different types of nucleic acids...
June 6, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28578643/glioblastoma-targeted-gene-therapy-based-on-pegfp-p53-loaded-superparamagnetic-iron-oxide-nanoparticles
#19
Touba Eslaminejad, Seyed Noureddin Nematollahi-Mahani, Mehdi Ansari
BACKGROUND: Bloodâ€"brain barrier (BBB) separates the neural tissue from circulating blood because of its high selectivity. This study focused on the in vitro application of magnetic nanoparticles to deliver Tp53 as a gene of interest to glioblastoma (U87) cells across a simulated BBB model that comprised KB cells. MATERIAL AND METHOD: After magnetic and non-magnetic nanoparticles were internalized by KB cells, their location in these cells was examined by transmission electron microscopy...
June 4, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28552050/readthrough-of-scn5a-nonsense-mutations-r1623x-and-s1812x-question-gene-therapy-in-brugada-syndrome
#20
Siyong Teng, Jian Huang, Zhan Gao, Jie Hao, Yuejin Yang, Shu Zhang, Jielin Pu, Rutai Hui, Yongjian Wu, Zheng Fan
PURPOSE: Readthrough of nonsense mutation are used as gene-specific treatment in some genetic disease. Response to readthrough treatment is determined by readthrough efficiency of various nonsense mutations. In this manuscript, we aimed to explore the harmful effect of nonsense mutation suppressions. METHODS: HEK293 cells were transfected with two SCN5A (encode cardiac Na+ channel) nonsense mutations, p.R1623X and p.S1812X. We applied two readthrough-enhancing methods (either aminoglycosides or by a siRNA targeting eukaryotic release factor eRF3a (a GTPase that binds eRF1)) to suppress these SCN5A nonsense mutations...
May 28, 2017: Current Gene Therapy
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