Current gene therapy
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Current gene therapy · Jan 2021
ReviewExploring the Role of Gene Therapy for Neurological Disorders.
Gene therapy is one of the frontier fields of medical breakthroughs that poses as an effective solution to previously incurable diseases. The delivery of the corrective genetic material or a therapeutic gene into the cell restores the missing gene function and cures a plethora of diseases, incurable by the conventional medical approaches. This discovery holds the potential to treat many neurodegenerative disorders such as muscular atrophy, multiple sclerosis, Parkinson's disease (PD) and Alzheimer's disease (AD), among others. ⋯ However, the use of gene therapy is only possible after procuring the in-depth knowledge of the immuno-pathogenesis and molecular mechanism of the disease. The process of gene therapy can be broadly categorized into three main steps: elucidating the target gene, culling the appropriate vector, and determining the best mode of transfer; each step mandating pervasive research. This review aims to dissertate and summarize the role, various vectors and methods of delivery employed in gene therapy with special emphasis on therapy directed at the central nervous system (CNS) associated with neurodegenerative diseases.
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Current gene therapy · Jan 2020
ReviewFeasibility of Mesenchymal Stem Cell Therapy for COVID-19: A Mini Review.
Patients infected with SARS-CoV-2 carry the coronavirus disease 2019 (COVID-19) which involves multiple systems and organs with acute respiratory distress syndrome (ARDS) as the most common complication, largely due to cytokine storms or dysregulated immunity. As such, there are many severe patients with complications such as cytokine storm syndrome (CSS), who have a high fatality rate. Neither specific anti-SARS-CoV-2 drugs nor vaccines exist currently. ⋯ In addition, MSCs have a strong ability to repair tissue damage and reduce the risk of severe complications such as acute lung injury and ARDS, and hopefully, reduce the fatality rate in these patients. There are several clinical types of research completed for treating COVID-19 with MSCs, all reporting restoration of T cells and clinical safety. Here we discuss the clinical prospect and conclude the therapeutic effects and potential mechanism for MSCs in treating COVID-19.
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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. ⋯ ERT has shown to be associated to a significant reduction of Gb3 accumulation in several tissues, in particular heart and kidney; moreover it improves pain related quality of life. Next generation lysosomal storage disorder treatment is based on new strategic approaches as stem cell based therapy, pharmacological chaperones, viral gene therapy; concerning Fabry's disease, it has been recently addressed to great interest this last innovative method, that is to say viral gene therapy, for delivering recombination enzyme into main involved tissues; promising results have been reported in animal models. Great efforts have been made and are still required in this field in order to make available a more effective, safer, advantageous therapeutic strategy for patients with Fabry's disease.
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Current gene therapy · Jan 2017
Sustained ELABELA Gene Therapy in High Salt-Induced Hypertensive Rats.
Elabela (ELA) is a recently identified apelin receptor agonist essential for cardiac development, but its biology and therapeutic potential are unclear. In humans, ELA transcripts are detected in embryonic stem cells, induced pluripotent stem cells, kidney, heart and blood vessels. ELA through the apelin (APJ) receptor promotes angiogenesis in vitro, relaxes murine aortic blood vessels and attenuates high blood pressure in vivo. The APJ receptor when bound to its original ligand, apelin, exerts peripheral vasodilatory and positive inotropic effects, conferring cardioprotection in vivo. ⋯ ELA is constitutively expressed in renal collecting ducts in rats. Sustained AAV-ELA expression may offer a potential long-term therapy for hypertension and renal remodeling.
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Current gene therapy · Jan 2017
ReviewA New Era for Hemoglobinopathies: More Than One Curative Option.
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. ⋯ Importantly, during the last years, additional curative options for patients with thalassemia and sickle cell disease are being developed, based on the ability to manipulate the genome by employing programmable nucleases and next-generation genome-modifying tools, thus providing the exciting prospects of targeted in-situ gene correction. In this review, we will summarize current developments in the new era of treatment for hemoglobinopathies, elaborate on lessons gained from gene therapy trials and discuss the exciting prospects and challenges of genome editing.