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  • Gene Therapy Treats Usher Syndrome Patients | 黄 义 基 金 会
    help usher syndrome patien Irv Arons Last week the Foundation Fighting Blindness and Oxford BioMedica announced that the first Usher Syndrome patient is about to be treated at Casey Eye Institute OHSU with Oxford s UshStat gene therapy treatment The trial is aimed at patients with Usher Syndrome Type 1B a form or retinitis pigmentosa that leads to blindness and hearing loss There is currently no treatment for this disease

    Original URL path: http://huange.org/web6/zh-hans/node/142 (2016-02-16)
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  • Gene Therapy Treats Usher Syndrome Patients | 黃 義 基 金 會
    and Oxford BioMedica announced that the first Usher Syndrome patient is about to be treated at Casey Eye Institute OHSU with Oxford s UshStat gene therapy treatment The trial is aimed at patients with Usher Syndrome Type 1B a form or retinitis pigmentosa that leads to blindness and hearing loss There is currently no treatment for this disease Based on laboratory pre clinical studies the researchers believe that a single

    Original URL path: http://huange.org/web6/zh-hant/node/142 (2016-02-16)
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  • Gene Therapy Treats Usher Syndrome Patients | Huange Foundation
    Fighting Blindness and Oxford BioMedica announced that the first Usher Syndrome patient is about to be treated at Casey Eye Institute OHSU with Oxford s UshStat gene therapy treatment The trial is aimed at patients with Usher Syndrome Type 1B a form or retinitis pigmentosa that leads to blindness and hearing loss There is currently no treatment for this disease Based on laboratory pre clinical studies the researchers believe that

    Original URL path: http://huange.org/web6/node/142?destination=node%2F142 (2016-02-16)
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  • QLT Receives Fast Track Designations for QLT091001 to Treat Both Leber Congenital Amaurosis and Retinitis Pigmentosa | Huange Foundation
    Retinitis Pigmentosa Wed 09 14 2011 08 05 sfan Check out this news VANCOUVER British Columbia Sep 14 2011 GlobeNewswire via COMTEX QLT Inc QLTI 4 60 CA QLT 3 90 today announced the Company s oral synthetic retinoid for retinal diseases QLT091001 has been granted two Fast Track designations by the U S Food and Drug Administration FDA for the treatment of Leber Congenital Amaurosis LCA due to inherited

    Original URL path: http://huange.org/web6/node/64?destination=node%2F64 (2016-02-16)
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  • Genable Technologies – a gene based medicines platform | Huange Foundation
    diseases based on the pioneering work of Professor Jane Farrar Dr Paul Kenna Professor Peter Humphries Genable utilizes well established clinically safe effective AAV vectors to obtain expression of RNA interference RNAi molecules which suppress the expression of both the faulty and normal gene copies and replaces this with a gene subtly altered to become refractory to suppression but still encoding a normal wild type protein The combination of suppression and replacement S R overcomes the significant hurdle in dominant disease of mutation variability by eliminating the need to target specific mutations in a wide range of disorders Genable s technology is protected by a broad suite of granted patents and patent applications in the USA EU and worldwide Genable s first gene medicine GT038 is for treatment of patients with rhodopsin RHO linked autosomal dominant retinitis pigmentosa adRP a debilitating form of inherited blindness resulting from a diverse array of mutations in the RHO gene This sub type of adRP affects approximately 1 in 30 000 people and represents an already identified and potentially treatable population of around 30 000 patients in the US and Europe Whilst we estimate the market opportunity for GT038 in this sub type of

    Original URL path: http://huange.org/web6/node/63?destination=node%2F63 (2016-02-16)
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  • Forums | Huange Foundation
    login Username Password Create new account Request new password Text Size Increase Normal Decrease Current Size 93 Languages 简体中文 繁體中文 English Home Forums Forums The text size have been saved as 100 The text size have been saved as 100 The text size have been saved as 93 Login to post new content in the forum Topic Replies Created Last reply Gene Therapy Successful in Usher Syndrome Type 2D Model

    Original URL path: http://huange.org/web6/forum/14 (2016-02-16)
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  • Gene Therapy Successful in Usher Syndrome Type 2D Model | Huange Foundation
    treatment into a clinical trial Usher syndrome is an inherited condition that causes defects in the function of cilia tiny hair like structures that act like a transportation system for proteins and biochemicals essential to the proper functioning of photoreceptors and cells of the inner ear In people with USH 2D a genetic defect leads to dysfunction of the cilia and ultimately vision and hearing loss Led by Dr Jun Yang the Foundation funded research team developed a safe manmade virus to deliver copies of normal USH 2D genes to the photoreceptors of the affected mice The treatment was injected under the retina where it was absorbed by the photoreceptors Tests revealed that copies of the therapeutic USH 2D gene reached photoreceptors and restored function of the cilia The virus used for therapy delivery an adeno associated virus or AAV is similar to AAVs used in other gene therapy lab studies and clinical trials One example is the series of landmark clinical trials that have restored some vision in more than 40 children and young adults with the early onset retinal disease known as Leber congenital amaurosis Dr Yang said that while the treatment restored ciliary function retinal degeneration and

    Original URL path: http://huange.org/web6/node/62?destination=node%2F62 (2016-02-16)
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  • 第16届视网膜国际大会有关RP的文章 | 黃 義 基 金 會
    干细胞替代受损视网膜细胞 由于干细胞是多能性的 所以可以分化成多种不同类型的细胞 干细胞分为胚胎干细胞 ES 及诱导性多潜能干细胞 iPS 后者产生于成熟细胞 干细胞疗法的目的就是替换那些受损的细胞 最近科学工作者已经发现 的体细胞 比如皮肤细胞 可以在试管内 体外 诱导生成为可变为视神经细胞的干细胞 这项重大发现可以使患有视网膜疾病的患者将体内成熟的细胞转换为多潜能细胞 这些多潜能细胞可以行使正常视神经细胞的功能 每年的视觉与眼年会中 都会有一些话题引起人们的注意 干细胞研究以及干细胞治疗方法在此次年会中引起了广泛的关注 多家实验室已经制定出了将胚胎干细胞 ES 及诱导性多潜能干细胞 iPS 转变为视网膜细胞的计划 并且通过动物实验发现 从上述两种干细胞转换成的视网膜细胞可以变为视网膜的一部分 在美国 基于干细胞技术的治疗产品都要通过美国食品 管理局的生物制品评价及研究中心审核 审核中要考虑的因素包括干细胞移植物的生命力 如何将干细胞输送到患者体内 干细胞进一步分化增殖成其他类型的细胞及癌变的可能性 以及细胞在体内的融合性等等 干细胞治疗是一门新兴的学科 因此在用于人体之前要对其安全性及效能进行完全彻底的研究 T A Reh K Wallace D O Clegg M Friedlander D W Fink 法国RP患者的EYS基因突变 研究人员通过对法国RP患者的研究发现 在法国并且可能在世界其他地方 EYS基因突变是导致RP的重要原因 EYS基因是人眼中已知的最大的基因 范围超过2兆位 在对超过200个散发及常染色体隐性RP患者的研究中 研究人员发现超过37例可能的EYS基因突变 一些患者只有单独突变 而其他患者有多种突变 这些患者中大多数都有RP的典型症状 比如中心视力受损较晚 在对上述患者的研究中发现EYS基因突变非常普遍 这表明在其他RP患者中 EYS基因突变可能也会扮演重要的角色 睫状营养因子疗法增加了黄斑厚度 王涛及她的同事在2010的年会上阐述了她们研究的新发现 她们的眼内移植封装细胞技术 ECT 的移植体内装有经过基因修改可以潜入眼内的睫状营养因子 CNTF

    Original URL path: http://huange.org/web6/zh-hant/node/57 (2016-02-16)
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