Destination Penis? Erectile Dysfunction As Possible Future Indication Of Therapeutic Gene Delivery.
Abstract 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...
Source: Current Gene Therapy - July 29, 2018 Category: Genetics & Stem Cells Authors: Gur S, Abdel-Mageed AB, Sikka SC, Bartolome AR, Hellstrom WJG Tags: Curr Gene Ther Source Type: research

Phenotypic characterization of a family with an in-frame deletion in the DMD gene and variable penetrance.
Abstract 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. Asymmetric left ventricular hypertrophy and a long QT interval were evidenced at the age of 1...
Source: Current Gene Therapy - July 9, 2018 Category: Genetics & Stem Cells Authors: Perez-Sanchez I, Sabater-Molina M, Rocamora MEN, Glover G, Escudero F, de Mingo Casado P, Gimeno-Blanes JR Tags: Curr Gene Ther Source Type: research

Current Advances in Small Activating RNAs for Gene Therapy: Principles, Applications and Challenges.
Abstract 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 reports have provided the possible ones to explain it. Furthermore, mounting evidences exhibit that saRNAs not only provide a new approa...
Source: Current Gene Therapy - June 19, 2018 Category: Genetics & Stem Cells Authors: Zhou LY, He ZY, Xu T, Wei YQ Tags: Curr Gene Ther Source Type: research

Inhibition of West Nile virus Replication by bifunctional siRNA targeting the NS2A and NS5 Conserved region.
CONCLUSION: Thus this bifunctional siRNA intervention paves the new way for therapeutic treatment of WNV disease. PMID: 29874999 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 6, 2018 Category: Genetics & Stem Cells Authors: Karothia D, Dash PK, Parida MM, Bhagyawant S, Kumar JS Tags: Curr Gene Ther Source Type: research

Preface: Translational Gene Therapy Coming of Age!
PMID: 29769000 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 19, 2018 Category: Genetics & Stem Cells Authors: Chang LJ, Lewin AS Tags: Curr Gene Ther Source Type: research

Editorial: Current Gene Therapy on Non-viral Gene Delivery and Therapy.
PMID: 29769001 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 19, 2018 Category: Genetics & Stem Cells Authors: Peng L Tags: Curr Gene Ther Source Type: research

AAV-mediated overexpression of IL-10 mitigates the inflammatory cascade in stimulated equine chondrocyte pellets.
CONCLUSIONS: Overexpression of IL-10 modulates the inflammatory response in chondrocytes, which may mitigate some of the deleterious effects of pro-inflammatory cascades in the posttraumatic joint. AAV5-IL-10 led to efficient and sustained overexpression of IL-10 in chondrocytes and could represent a viable treatment option for preventing osteoarthritis. PMID: 29749312 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 10, 2018 Category: Genetics & Stem Cells Authors: Ortved K, Begum L, Stefanovski D, Nixon A Tags: Curr Gene Ther Source Type: research

Editorial: Genetics and Gene Therapy of Lysosomial Storage Disorders.
PMID: 29741137 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 9, 2018 Category: Genetics & Stem Cells Authors: Tuttolomondo A Tags: Curr Gene Ther Source Type: research

miR-7 replacement therapy in Parkinson's disease.
Abstract 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 the 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 was related to α-Syn accumulation, loss of dop...
Source: Current Gene Therapy - April 30, 2018 Category: Genetics & Stem Cells Authors: Titze-de-Almeida R, Titze-de-Almeida SS Tags: Curr Gene Ther Source Type: research

Transplantation of BDNF Gene Recombinant Mesenchymal Stem Cells and Adhesive Peptide-modified Hydrogel Scaffold for Spinal Cord Repair.
In this study, gene transfection technology was applied to prepare BDNF gene recombinant MSCs based on our previously reported liposomal vector ScreenFect® A. To improve cellular survival and gene expression after in situ implantation of MSCs, an adhesive peptide modified hydrogel scaffold was constructed using hyaluronic acid. The scaffold was optimized and modified with an adhesive peptide PPFLMLLKGSTR. The transfected MSCs exhibited improved cellular survival and sustained gene expression in the three-dimentional (3D) scaffold in vitro. Compared to untransfected MSCs, gene recombinant MSCs effectively improved spina...
Source: Current Gene Therapy - April 13, 2018 Category: Genetics & Stem Cells Authors: Li LM, Huang LL, Jiang XC, Chen JC, OuYang HW, Gao JQ Tags: Curr Gene Ther Source Type: research

Gene Therapy to Enhance Bone and Cartilage Repair in Orthopaedic Surgery.
Abstract 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. Tissue engineering via gene therapy attempts to provide an alternative treatment strategy to a...
Source: Current Gene Therapy - April 10, 2018 Category: Genetics & Stem Cells Authors: Bougioukli S, Evans C, Alluri R, Ghivizzani S, Lieberman J Tags: Curr Gene Ther Source Type: research

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.
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. Percutaneous endocardial Ad-HGF gene transfer was injected with a catheter-based intramyocardial delivery system in the experimental group. Safety parameters were measured and compared between baseline and follow-ups i...
Source: Current Gene Therapy - April 4, 2018 Category: Genetics & Stem Cells Authors: Meng H, Chen B, Tao Z, Xu Z, Wang L, Weizhu J, Hong Y, Liu X, Wang H, Wang L, Wu Z, Yan Z Tags: Curr Gene Ther Source Type: research

Genetics and Therapies for GM2 Gangliosidosis.
Abstract 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 ...
Source: Current Gene Therapy - April 4, 2018 Category: Genetics & Stem Cells Authors: Cachon-Gonzalez MB, Zaccariotto E, Cox TM Tags: Curr Gene Ther Source Type: research

Genetics and gene therapy of Anderson-Fabry disease.
Abstract 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 typin...
Source: Current Gene Therapy - April 4, 2018 Category: Genetics & Stem Cells Authors: Simonetta I, Tuttolomondo A, Di Chiara T, Miceli S Tags: Curr Gene Ther Source Type: research

Genetics and Gene therapy in Hunter disease.
Abstract 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 a...
Source: Current Gene Therapy - April 4, 2018 Category: Genetics & Stem Cells Authors: Simona S, Falvo F, Apa R, Pensabene L, Bonapace G, Moricca MT, Concolino D Tags: Curr Gene Ther Source Type: research

Dysfunction in Brain-Derived Neurotrophic Factor Signaling Pathway and Susceptibility to Schizophrenia, Parkinson's and Alzheimer's Diseases.
Abstract 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. Transcriptio...
Source: Current Gene Therapy - March 2, 2018 Category: Genetics & Stem Cells Authors: Mohammadi A, Amooeian VG, Rashidi E Tags: Curr Gene Ther Source Type: research

Application of Optogenetics in Gene Therapy.
Abstract 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. Here, we summarize rep...
Source: Current Gene Therapy - March 2, 2018 Category: Genetics & Stem Cells Authors: Kushibiki T, Ishihara M Tags: Curr Gene Ther Source Type: research

Applications And Prospects Of Non-Viral Vectors In Bone Regeneration.
Abstract 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. Gene therapy offers an alternative method to cure bone...
Source: Current Gene Therapy - February 27, 2018 Category: Genetics & Stem Cells Authors: Yang W, Wang F, Feng L, Yan S, Guo R Tags: Curr Gene Ther Source Type: research

CRISPR-Cas System as a Genome Engineering Platform: Applications in Biomedicine and Biotechnology.
Abstract 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. Next, the applications of CRISPR-Cas9 in various ways, such as efficient generation of a wide varie...
Source: Current Gene Therapy - February 20, 2018 Category: Genetics & Stem Cells Authors: Hashemi A Tags: Curr Gene Ther Source Type: research

Whats ´New In Gene Therapy Of Hemophilia.
CONCLUSION: Achieving a safe and efficient remedy for hemophilia A and B by means of GT vector engineering needs further improvement. No randomized or quasi-randomized clinical trials of GT for hemophilia have been found. Given it is in its incipient period, there is need for well-designed clinical trials to evaluate the long-term practicability, efficacy and risks of GT for PWH. PMID: 29446741 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - February 14, 2018 Category: Genetics & Stem Cells Authors: Rodriguez-Merchan EC Tags: Curr Gene Ther Source Type: research

Current Understanding Of Inflammatory Responses In Acute Kidney Injury.
Abstract Acute kidney injury has been a tough complex with increased mortality and morbidity. Inflammatory responses, including innate and adaptive immune responses, involve in the initiation and development of acute kidney injury, especially under the ischemic circumstances. Tubular cells and distinct immune cell subgroups play a critical role in the pathogenesis of inflammation. Current gene therapies show their benefits in renal repair. Here, we reviewed the renal inflammatory infiltration, inflammatory mediators, oxidative stress and potential signaling pathways, which give rise to the kidney diseases, in the ...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Hu C, Sheng Y, Qian Z Tags: Curr Gene Ther Source Type: research

CX3CL1/CX3CR1 axis, as The Therapeutic Potential In Renal Diseases: Friend Or Foe?
Abstract 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 d...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Zhuang Q, Cheng K, Ming Y Tags: Curr Gene Ther Source Type: research

Epigenetic Regulation Of Regulatory T Cells In Kidney Disease And Transplantation.
Abstract 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 ce...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Liu Y, Peng B, Wu S, Xu N Tags: Curr Gene Ther Source Type: research

Gene Therapy In Kidney Transplantation: Evidence Of Efficacy And Future Directions.
Abstract 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. Recent studies have successfully attempted to use gene therapy to target the key molecules involved in immune responses during transplantation. This strategy has the potential to ...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Li J, Qi G, Tu G, Yang C, Rong R Tags: Curr Gene Ther Source Type: research

Gene-Modified Mesenchymal Stem Cell-Based Therapy In Renal Ischemia-Reperfusion Injury.
Abstract 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. MSC-based therapy has drawn wide attention for its therapeutic potential in renal IRI. The admi...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Xu H, Chen C, Hu L, Hou J Tags: Curr Gene Ther Source Type: research

How Do Dendritic Cells Play The Role In Ischemia/Reperfusion Triggered Kidney Allograft Rejection.
Abstract 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 a...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Cai S, Ichimaru N, Takahara S Tags: Curr Gene Ther Source Type: research

Role of Complement Properdin in Renal Ischemia-Reperfusion Injury.
Abstract 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. Nevertheless, the role of complement properdin, the sole positive regulator of the alternative pathway, in IRI-induced AKI has not been well defined. This revi...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Zwaini Z, Dai H, Stover C, Yang B Tags: Curr Gene Ther Source Type: research

Role of Immune Cells in Diabetic Kidney Disease.
Abstract 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. This overview of macrophages, dendritic cells, T lymphocytes, B lymphocytes, neutrophils and mast cells is closely involved in the pathologic process of DKD...
Source: Current Gene Therapy - February 13, 2018 Category: Genetics & Stem Cells Authors: Yang X, Mou S Tags: Curr Gene Ther Source Type: research

Dosage and Passage Dependent Neuroprotective Effects of Exosomes Derived from Rat Bone Marrow Mesenchymal Stem Cells: An In Vitro Analysis.
CONCLUSION: Our study suggests that adult stem cells derived exosomes could be a potential therapeutic agent to confer neuroprotection in neurodegenerative diseases like Alzheimer's disease. PMID: 29366415 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - January 24, 2018 Category: Genetics & Stem Cells Authors: Venugopal C, Shamir C, Senthilkumar S, Babu JV, Sonu PK, Nishtha KJ, Rai KS, Shobha K, Dhanushkodi A Tags: Curr Gene Ther Source Type: research

A New Era For Hemoglobinopathies: More Than One Curative Options.
Abstract Hemoglobinopathies, including severe β-thalassemia and sickle cell disease, represent the most common monogenic disorders worldwide. Allogeneic hematopoietic stem cell transplantation (allo-HCT) is the only approved curative option for these syndromes, albeit limited to patients having a suitable donor. Gene therapy, by making use of the patient's own hematopoietic stem cells to introduce a normal copy of the β-globin gene by viral vectors, bridged the gap between the need for cure of patients with hemoglobinopathies and the lack of a donor, without incurring the immunological risks of allo-HSCT...
Source: Current Gene Therapy - January 19, 2018 Category: Genetics & Stem Cells Authors: Psatha N, Papayanni PG, Yannaki E Tags: Curr Gene Ther Source Type: research

Neurodegenerative Disorders Treatment: The MicroRNA Role.
Abstract 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 intens...
Source: Current Gene Therapy - January 19, 2018 Category: Genetics & Stem Cells Authors: Ridolfi B, Abdel-Haq H Tags: Curr Gene Ther Source Type: research

Nonviral Delivery Systems For Cancer Gene Therapy: Strategies And Challenges.
Abstract 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. In this review, we will...
Source: Current Gene Therapy - January 19, 2018 Category: Genetics & Stem Cells Authors: Shim G, Kim D, Le QV, Park GT, Kwon T, Oh YK Tags: Curr Gene Ther Source Type: research

Molecular Adjuvants Based On Plasmids Encoding Protein Aggregation Domains Affect Bone Marrow Niche Homeostasis.
CONCLUSION: The above findings provide compelling support to the thesis that adjuvants based on plasmids encoding protein aggregation domains disrupt the physiological features of the bone marrow elements. PMID: 29303078 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - January 5, 2018 Category: Genetics & Stem Cells Authors: Sabbieti MG, Lacava G, Amaroli A, Marchetti L, Censi R, Di Martino P, Agas D Tags: Curr Gene Ther Source Type: research

The Progress of Gene Therapy for Leber`s Optic Hereditary Neuropathy.
Abstract Leber's Optic Hereditary Neuropathy (LHON) is a common cause of teenaged blindness in both eyes for which there is currently no effective treatment. In 1871, the German ophthalmologist Theodor Leber was the first to describe the clinical characteristics of his namesake disease, and through unremitting efforts over the past 100 years, researchers have continued to increase their understanding of LHON. In recent years, using gene therapy, several groups have obtained breakthroughs in the treatment of the disease. In this article, we will review the challenging journey that researchers faced towards our curr...
Source: Current Gene Therapy - November 29, 2017 Category: Genetics & Stem Cells Authors: Zhang Y, Tian Z, Yuan J, Liu C, Liu HL, Ma SQ, Li B Tags: Curr Gene Ther Source Type: research

CRISPR/Cas9 Editing to Facilitate and Expand Drug Discovery.
CONCLUSION: We review the literature and hurdles that have been overcome to develop the current generation of tools being used to enrich the drug discovery paradigm. PMID: 29173168 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 21, 2017 Category: Genetics & Stem Cells Authors: Robert F, Huang S, Pelletier J Tags: Curr Gene Ther Source Type: research

CRISPR/Cas9 Gene Editing: From Basic Mechanisms To Improved Strategies For Enhanced Genome Engineering In Vivo.
Abstract Targeted genome editing using the CRISPR/Cas9 technology is becoming a major area of research due to its high potential for the treatment of genetic diseases. Our understanding of this approach has expanded in recent years yet several new challenges have presented themselves as we explore the boundaries of this exciting new technology. Chief among these is improving the efficiency but also the preciseness of genome editing. The efficacy of CRISPR/Cas9 technology relies in part on the use of one of the major DNA repair pathways, Homologous recombination (HR), which is primarily active in S and G2 phases of...
Source: Current Gene Therapy - November 21, 2017 Category: Genetics & Stem Cells Authors: Salsman J, Masson JY, Orthwein A, Dellaire G Tags: Curr Gene Ther Source Type: research

First-In-Human Phase 1 CRISPR Gene Editing Cancer Trials: Are We Ready?
Abstract A prospective first-in-human Phase 1 CRISPR gene editing trial in the United States for patients with melanoma, synovial sarcoma, and multiple myeloma offers hope that gene editing tools may usefully treat human disease. An overarching ethical challenge with first-in-human Phase 1 clinical trials, however, is knowing when it is ethically acceptable to initiate such trials on the basis of safety and efficacy data obtained from pre-clinical studies. If the pre-clinical studies that inform trial design are themselves poorly designed - as a result of which the quality of pre-clinical evidence is deficient - t...
Source: Current Gene Therapy - November 21, 2017 Category: Genetics & Stem Cells Authors: Baylis F, McLeod M Tags: Curr Gene Ther Source Type: research

New Developments in CRISPR/Cas-based Functional Genomics and their Implications for Research using Zebrafish.
Abstract Genome editing using CRISPR/Cas9 has advanced very rapidly in its scope, versatility and ease of use. Zebrafish (Danio rerio) has been one of the vertebrate model species where CRISPR/Cas9 has been applied very extensively for many different purposes and with great success. In particular, disease modeling in zebrafish is useful for testing specific gene variants for pathogenicity in a preclinical setting. Here we describe multiple advances in diverse species and systems that can improve genome editing in zebrafish. To achieve temporal and spatial precision of genome editing, many new technologies can be a...
Source: Current Gene Therapy - November 21, 2017 Category: Genetics & Stem Cells Authors: Prykhozhij SV, Caceres L, Berman JN Tags: Curr Gene Ther Source Type: research

Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.
CONCLUSIONS: Restoration of the wild type and shorter form of dystrophin highlights the therapeutic potential of CRISPR technology for DMD. PMID: 29173172 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 21, 2017 Category: Genetics & Stem Cells Authors: Wong TWY, Cohn RD Tags: Curr Gene Ther Source Type: research

Gene Therapy And Retinal Diseases.
Conclusion Gene therapy represents an emerging and promising therapeutic approach for the treatment not only of rare inherited retinal diseases but also much more common retinal pathologies. PMID: 29149824 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 16, 2017 Category: Genetics & Stem Cells Authors: Campa C, Gallenga CE, Bolletta E, Perri P Tags: Curr Gene Ther Source Type: research

Cardioprotective Effects Of Serca2a Overexpression Against Ischemia-Reperfusion-Induced Injuries In Rats.
CONCLUSION: Our findings demonstrated that the overexpression of Serca2a plays an important role in myocardial protection from I/R injury and postischemic functional recovery, which may be via anti-necrotic, anti-apoptotic and pro-autophagy signal pathways. Our research provides solid basic data and new perspective for clinical treatment in heart failure patients with long-term over-expression of Serca2a. PMID: 29141547 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 10, 2017 Category: Genetics & Stem Cells Authors: Jian Y, Tian LL, Wang LH, Zhao XD, Chen JW, Murao K, Zhu W, Dong L, Wang GQ, Sun WP, Zhang GX Tags: Curr Gene Ther Source Type: research

Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders.
CONCLUSION: This review highlights several key factors to be taken into consideration to design successful preclinical and clinical gene therapy experiments with respect to the vehicle of delivery and the route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotide therapy and recent clinical trial developments. PMID: 29034834 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 13, 2017 Category: Genetics & Stem Cells Authors: Donde A, Wong PC, Chen LL Tags: Curr Gene Ther Source Type: research

Effect of Oral Nonionic Polymeric Micelles Delivered Parathyroid Hormone cDNA on Bone Density and Microarchitecture.
CONCLUSION: Results showed that bone mineral density, bone volume fraction, and trabecular thickness were significantly increased in Group 1 over time, compared with those in Group 2 and Group 3. In conclusion, significantly improved bone mineral density and bone microstructure were observed in ovariectomized rats treated with PTH (1-34) cDNA delivered by nonionic polymeric micelles. PMID: 28982328 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 4, 2017 Category: Genetics & Stem Cells Authors: Chen SH, Ko JY, Chou FF Tags: Curr Gene Ther Source Type: research

Comparative Study Of Adeno-Associated Virus, Adenovirus, Baculovirus And Lentivirus Vectors For Gene Therapy Of The Eyes.
Conclusion Our findings show that intravitreal gene delivery is safe and feasible with AAV, AdV and lentivirus vectors. PMID: 28982327 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 3, 2017 Category: Genetics & Stem Cells Authors: Kalesnykas G, Kokki E, Alasaarela L, Lesch HP, Tuulos T, Kinnunen K, Uusitalo H, Airenne K, Ylä-Herttuala S Tags: Curr Gene Ther Source Type: research

Editorial: Gene Therapy for Fanconi Anemia Enters a New Clinical Era.
PMID: 28929955 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 22, 2017 Category: Genetics & Stem Cells Authors: Verhoeyen E Tags: Curr Gene Ther Source Type: research

MicroRNAs Mediated MMP Regulation: Current Diagnostic and Therapeutic Strategies for Metabolic Syndrome.
Abstract Metabolic syndrome (MS) is a global socioeconomic problem rapidly progressing in accordance with increasing body mass index (BMI) and age. It is a consortium of risk factors, such as dyslipidaemia, insulin resistance, leptin resistance, reduced adiponectin, glucose intolerance, hyperglycemia, and hypertension. Collectively, these factors accelerate the onset of type 2 diabetes mellitus, cardiovascular disease, stroke, and certain cancers such as breast, liver pancreatic, and colon cancer. Extracellular matrix (ECM) and basement membrane remodeling play a central role during pathogenesis of MS as they regu...
Source: Current Gene Therapy - July 7, 2017 Category: Genetics & Stem Cells Authors: Saxena S, Jain A, Rani V Tags: Curr Gene Ther Source Type: research

Non-viral Delivery Systems for breast cancer gene therapy.
CONCLUSION: In this review, we focus on recent advances in the intracellular delivery of DNA and siRNA to the cancer cells with emphasis on breast cancer. PMID: 28595562 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 6, 2017 Category: Genetics & Stem Cells Authors: Vaseghi G, Rafiee L, Javanmard SH Tags: Curr Gene Ther Source Type: research

Glioblastoma Targeted Gene Therapy based on pEGFP/p53-Loaded Superparamagnetic Iron Oxide Nanoparticles.
CONCLUSION: It could be concluded that a significant increase in total apoptosis was induced in cells by magnetic nanoparticles, coupled with exposure to a magnetic force (p ≤0.01) as compared with cells that were not exposed to magnetism. PMID: 28578643 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 4, 2017 Category: Genetics & Stem Cells Authors: Eslaminejad T, Nematollahi-Mahani SN, Ansari M Tags: Curr Gene Ther Source Type: research

Readthrough of SCN5A Nonsense Mutations R1623X and S1812X Question Gene-therapy in Brugada Syndrome.
CONCLUSIONS: These results demonstrated that readthrough-enhancing methods effectively suppressed nonsense mutations in SCN5A and restored the expression of full-length channels. But the restored channels may increase the risk of arrhythmia. The strategies for nonsense mutations suppression provides potential evidence for personalized medicine for the treatment of genetic disorders. PMID: 28552050 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 28, 2017 Category: Genetics & Stem Cells Authors: Teng S, Huang J, Gao Z, Hao J, Yang Y, Zhang S, Pu J, Hui R, Wu Y, Fan Z Tags: Curr Gene Ther Source Type: research

Editorial: Smart and Controllable Systems for Gene Delivery.
PMID: 28524008 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 17, 2017 Category: Genetics & Stem Cells Authors: Mostaghaci B Tags: Curr Gene Ther Source Type: research