Leber遗传性视神经病变的治疗或许不再是难题:GS010的REVERSE III期试验数据即将公布

2019-03-17 不详 网络

GenSight是一家专注于开发视网膜神经退行性疾病和中枢神经系统疾病创新基因疗法的生物制药公司,近日宣布GS010的REVERSE III期临床试验结果将于2019年3月16日至21日在第45届北美神经眼科学会(NANOS)年会上公布。

GenSight是一家专注于开发视网膜神经退行性疾病和中枢神经系统疾病创新基因疗法的生物制药公司,近日宣布GS010REVERSE III临床试验结果将于2019316日至21日在第45届北美神经眼科学会(NANOS)年会上公布。结果将由美国埃默里大学医学院眼科学和神经学教授Nancy J. Newman博士主讲,Newman博士是REVERSE试验的国际首席研究员,被广泛认为是Leber遗传性视神经病变(LHON)的主要权威之一。

GS010是由线粒体靶向序列(MTS)专有技术平台研发,主要针对Leber遗传性视神经病变(LHON)。该平台使用AAV载体(腺相关病毒)解决线粒体内的基因缺陷,将目的基因转移到待表达的细胞中并产生功能性蛋白质,然后通过特定的核苷酸序列将其转运至线粒体,以恢复缺失的线粒体功能。


原始出处:

http://www.firstwordpharma.com/node/1629954#axzz5iPvQruud

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    2019-10-17 anminleiryan
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  5. 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encryptionId=412b95155f8, topicName=遗传性视神经病变)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=55683, createdName=仁医06, createdTime=Thu Feb 20 04:33:00 CST 2020, time=2020-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2005528, encodeId=bac12005528b8, content=<a href='/topic/show?id=7e0315401a8' target=_blank style='color:#2F92EE;'>#reverse#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=40, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15401, encryptionId=7e0315401a8, topicName=reverse)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4b1f500, createdName=zengjiayao, createdTime=Wed Sep 18 21:33:00 CST 2019, time=2019-09-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1916089, encodeId=72001916089b6, content=<a href='/topic/show?id=c5a89550ff' target=_blank style='color:#2F92EE;'>#III#</a>, beContent=null, objectType=article, 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  6. 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authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/656F31B644588474D1C46903A7F84373/100, createdBy=4e842500199, createdName=ms24272190615788285182, createdTime=Sun Jul 28 15:33:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1491339, encodeId=f96d14913394e, content=<a href='/topic/show?id=5316e4387aa' target=_blank style='color:#2F92EE;'>#神经病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74387, encryptionId=5316e4387aa, topicName=神经病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=1ab48885684, createdName=axin012, createdTime=Tue Mar 19 09:33:00 CST 2019, time=2019-03-19, status=1, ipAttribution=)]
    2019-04-08 juliusluan78
  7. 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href='/topic/show?id=24ef96066d' target=_blank style='color:#2F92EE;'>#II期试验#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9606, encryptionId=24ef96066d, topicName=II期试验)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTKUdkK5dVTue7YQDvwqM1ugOuEpDZEzvXhoGClFiavehcrNAYDuPbks3MkKLPpyAEicgic80KguxwicYQ/132, createdBy=ec952500165, createdName=ms5187542471278033, createdTime=Sun Nov 17 16:33:00 CST 2019, time=2019-11-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2055262, encodeId=a7ed205526227, content=<a href='/topic/show?id=6f9391e7414' target=_blank style='color:#2F92EE;'>#试验数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=91774, encryptionId=6f9391e7414, topicName=试验数据)], attachment=null, authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/656F31B644588474D1C46903A7F84373/100, createdBy=4e842500199, createdName=ms24272190615788285182, createdTime=Sun Jul 28 15:33:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1491339, encodeId=f96d14913394e, content=<a href='/topic/show?id=5316e4387aa' target=_blank style='color:#2F92EE;'>#神经病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74387, encryptionId=5316e4387aa, topicName=神经病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=1ab48885684, createdName=axin012, createdTime=Tue Mar 19 09:33:00 CST 2019, time=2019-03-19, status=1, ipAttribution=)]
    2020-02-08 gj0740
  8. 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  9. 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    2019-03-19 axin012

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GenSight是一家专注于发现和开发视网膜神经退行性疾病和中枢神经系统疾病创新基因疗法的生物制药公司。GenSight生物技术公司将于2018年10月27日至30日在伊利诺伊州芝加哥举行的2018年美国眼科学会年会上,公布GS010的REVERSE III期临床试验的积极数据。