Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies.

Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies. Adv Exp Med Biol. 2019 Nov 15;: Authors: Şişli HB, Hayal TB, Seçkin S, Şenkal S, Kıratlı B, Şahin F, Doğan A Abstract The identification of human embryonic stem cells and reprogramming technology to obtain induced pluripotent stem cells from adult somatic cells have provided unique opportunity to create human disease models, gene editing strategies and cell therapy options.Development of pluripotent stem cells from somatic cells and genomic manipulation tools enabled to use site specific nucleases in the cell therapy research. Identification of efficient gene manipulation, safe differentiation and use will provide a novel strategy to treat many diseases in the near future. Current available registered clinical trials clearly indicate the need for pluripotent stem cell and gene therapy treatment options. Although gene editing based pluripotent stem cell research is a popular field for research worldwide, improvement of clinical approaches for treatment still remains to be investigated. In this review, we summarized the current situation of gene editing based pluripotent cell therapy developments and applications in clinics. PMID: 31728915 [PubMed - as supplied by publisher]
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research

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Source: Stem Cell Research and Therapy - Category: Stem Cells Authors: Tags: Research Source Type: research
AbstractPurpose of reviewTo present an updated appraisal of hematopoietic stem cell transplant (HSCT) and gene therapy for X-linked adrenoleukodystrophy (ALD) in the setting of a novel, presymptomatic approach to disease.Recent findingsOutcomes in HSCT for ALD have been optimized over time due to early patient detection, improved myeloablative conditioning regimens, and adjunctive treatment for patients with advanced cerebral disease. Gene therapy has arrested disease progression in a cohort of boys with childhood cerebral ALD. New therapeutic strategies have provided the clinical basis for the implementation of Newborn Sc...
Source: Current Treatment Options in Neurology - Category: Neurology Source Type: research
CONCLUSION: This provides a simplified protocol for isolating high-purity human CD34 + HSPCs from banked UCB adaptable to current Good Manufacturing Practice. This protocol reduces the number of steps and associated risks and thus total production costs. Importantly, the isolated CD34 + HSPCs possess in vivo repopulating activity in immunodeficient mice, making them a suitable starting population for ex vivo culture and gene editing. PMID: 31769050 [PubMed - as supplied by publisher]
Source: Transfusion - Category: Hematology Authors: Tags: Transfusion Source Type: research
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Source: Stem Cell Research - Category: Stem Cells Source Type: research
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Source: FEBS Letters - Category: Biochemistry Authors: Tags: Review Source Type: research
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Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
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