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GenSight to launch trial of gene therapy, visual stimulation device combo in UK

The UK’s Medicines and Healthcare Regulatory Agency has approved GenSight Biologics‘ (EPA:SIGHT) application to combine gene therapy and a wearable optronic visual stimulation device in patients with retinitis pigmentosa. The first-in-man Phase I/II trial is designed to study the safety and tolerability of GenSight’s GS030 combination therapy in three groups of three patients each. Get the full story at our sister site, Drug Delivery Business News. The post GenSight to launch trial of gene therapy, visual stimulation device combo in UK appeared first on MassDevice.
Source: Mass Device - Category: Medical Devices Authors: Tags: Clinical Trials Optical/Ophthalmic Pharmaceuticals Regulatory/Compliance Wall Street Beat gensightbiologics Source Type: news

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Authors: Chuang K, Fields MA, Del Priore LV Abstract The advent of gene editing has introduced the ability to make changes to the genome of cells, thus allowing for correction of genetic mutations in patients with monogenic diseases. Retinal diseases are particularly suitable for the application of this new technology because many retinal diseases, such as Stargardt disease, retinitis pigmentosa (RP), and Leber congenital amaurosis (LCA), are monogenic. Moreover, gene delivery techniques such as the use of adeno-associated virus (AAV) vectors have been optimized for intraocular use, and phase III trials are well un...
Source: The Yale Journal of Biology and Medicine - Category: Universities & Medical Training Tags: Yale J Biol Med Source Type: research
Authors: Yusuf IH, Shanks ME, Clouston P, MacLaren RE Abstract FLVCR1 (feline leukemia virus subgroup c receptor 1) is a transmembrane protein involved in the trafficking of intracellular heme. Homozygous variants in FLVCR1 have been described in association with a clinical syndrome of posterior column ataxia with retinitis pigmentosa (PCARP). Here, we describe a patient with non-syndromic retinitis pigmentosa homozygous for a splice-site variant in FLVCR1 (c.1092 + 5G>A) without evidence of posterior column ataxia or cerebellar degeneration. We suggest an association between intronic splice-site varia...
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
Human Gene Therapy Dec 2017, Vol. 28, No. 12: 1180-1188.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy Dec 2017, Vol. 28, No. 12: 1189-1201.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Condition:   Non-syndromic Retinitis Pigmentosa Intervention:   Combination Product: Gene therapy: GS030-DP AND Medical device: GS030-MD Sponsor:   GenSight Biologics Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Publication date: Available online 31 October 2017 Source:Progress in Retinal and Eye Research Author(s): Marina França Dias, Kwangsic Joo, Jessica A. Kemp, Silvia Ligório Fialho, Armando da Silva Cunha, Se Joon Woo, Young Jik Kwon Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people worldwide. It is characterized with progressive loss of rods and cones and causes severe visual dysfunction and eventual blindness in bilateral eyes. In addition to more than 3000 genetic mutations from about 70 genes, a wide genetic overlap with other types of retinal dystrophies has been ...
Source: Progress in Retinal and Eye Research - Category: Opthalmology Source Type: research
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