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Genetic Engineering Publications - GEG Tech top picks

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Genetic Engineering Publications - GEG Tech top picks

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Human ESC–derived retinal epithelial cell sheets potentiate rescue of photoreceptor cell loss in rats with retinal degeneration | Genetic Engineering Publications - GEG Tech top picks | Scoop.it

From stm.sciencemag.org - December 21, 2017 8:50 AM

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Human ESC–derived retinal epithelial cell sheets potentiate rescue of photoreceptor cell loss in rats with retinal degeneration

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Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells | Genetic Engineering Publications - GEG Tech top picks | Scoop.it

From www.nature.com - January 28, 2016 1:05 PM

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Here the authors used the CRISPR system to precisely repair in patient-specific iPSCs an RPGR point mutation that causes X-linked retinitis pigmentosa (XLRP). They observed that 13% of RPGR gene copies showed mutation correction and conversion to the wild-type allele. This important proof-of-concept finding supports the development of personalized iPSC-based transplantation therapies for retinal disease.

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From informahealthcare.com - January 29, 2015 5:07 AM

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This review discusses the wide-ranging applications of iPSCs and CRISPR with an ultimate view towards understanding how these two technologies can come together to address disease heterogeneity and orphan genes in a novel personalized medicine platform. An extensive literature search was conducted in PubMed and Google Scholar, with a particular focus on high-impact research published within the last 1 – 2 years and centered broadly on the subjects of retinal gene therapy, iPSC-derived outer retina cells, stem cell transplantation and CRISPR/Cas gene editing.

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In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration - Nature | Genetic Engineering Publications - GEG Tech top picks | Scoop.it

From www.nature.com - November 21, 2016 9:49 AM

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In this work, the devise a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and, more importantly, in vivo (for example, in neurons of postnatal mammals). As a proof of concept of its therapeutic potential, they demonstrate the efficacy of HITI in improving visual function using a rat model of the retinal degeneration condition retinitis pigmentosa. The HITI method presented here establishes new avenues for basic research and targeted gene therapies.

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From www.nature.com - August 17, 2015 10:15 AM

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In this study, the authors report successful generation of human induced pluripotent stem cells (iPSC) from skin fibroblasts of a patient harboring a novel Ser331Cysfs*5 mutation in the MERTK gene. Upon differentiation of these iPSC towards RPE, patient-specific RPE cells exhibited defective phagocytosis, a characteristic phenotype of MERTK deficiency observed in human patients and animal models.

This cellular model allows to investigate in detail the disease mechanism, explore screening of a variety of therapeutic compounds/reagents and design either combined cell and gene- based therapies or independent approaches.

www.geg-tech.com/Vectors

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Genetic Engineering Publications - GEG Tech top picks

Human ESC–derived retinal epithelial cell sheets potentiate rescue of photoreceptor cell loss in rats with retinal degeneration

Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells

Personalized therapeutic strategies for patients with retinitis pigmentosa

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration - Nature

Human iPSC derived disease model of MERTK-associated retinitis pigmentosa -