骨科

订阅

MedSci主页 / 所有栏目 / 骨科

MYO-101基因疗法治疗肢带肌营养不良症的积极结果

2019-02-28 不详 网络

MYO-101基因疗法肌营养不良症β-SG
关注

Sarepta制药公司本周三透露，2型肢带型肌营养不良症（LGMD2E）基因治疗候选药物MYO-101在3名患者的I / IIa期研究中展现了积极的阳性结果。

Sarepta制药公司本周三透露，2型肢带型肌营养不良症（LGMD2E）基因治疗候选药物MYO-101在3名患者的I / IIa期研究中展现了积极的阳性结果。Sarepta首席执行官Doug Ingram表示：“我们的基因治疗已经使患者产生了高水平的缺失蛋白质的稳定表达，并在Duchenne肌营养不良症和LGMD2E中产生了相关生物标志物的强烈结果”。

在该研究中，三名4岁至13岁的儿童在接受MYO-101输注约两个月后，所有患者都表现出β-sarcoglycan（β-SG）的强烈表达，且β-SG恰当地定位于肌肉肌膜。与正常对照相比，肌纤维膜中平均转导的β-SG蛋白表达为51％，而预先指定的成功率为20％。Sarepta指出同时，所有参与者均表现出血清肌酸激酶水平的显著下降，平均下降超过90％。


原始出处：

http://www.firstwordpharma.com/node/1627040#axzz5gj4KGYJT

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

版权声明：
本网站所有内容来源注明为“梅斯医学”或“MedSci原创”的文字、图片和音视频资料，版权均属于梅斯医学所有。非经授权，任何媒体、网站或个人不得转载，授权转载时须注明来源为“梅斯医学”。其它来源的文章系转载文章，或“梅斯号”自媒体发布的文章，仅系出于传递更多信息之目的，本站仅负责审核内容合规，其内容不代表本站立场，本站不负责内容的准确性和版权。如果存在侵权、或不希望被转载的媒体或个人可与我们联系，我们将立即进行删除处理。
在此留言
评论区 (5)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1737070, encodeId=7d951e37070cd, content=<a href='/topic/show?id=6ee1810e0ad' target=_blank style='color:#2F92EE;'>#肌营养不良#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81070, encryptionId=6ee1810e0ad, topicName=肌营养不良)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd6034244098, createdName=751943081_32627960, createdTime=Mon Oct 28 23:14:00 CST 2019, time=2019-10-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=368759, encodeId=d5fc368e59fa, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=5b172565856, createdName=12571e9dm53暂无昵称, createdTime=Mon Jul 01 16:08:32 CST 2019, time=2019-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007056, encodeId=68ac200e056b2, content=<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f7482500171, createdName=ms4468361851264128, createdTime=Thu Dec 19 07:14:00 CST 2019, time=2019-12-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=364204, encodeId=cefe36420437, content=这些伟大的科研人员是上帝派来的天使, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cdnapi.center.medsci.cn/medsci/head/2019/04/06/137a837e32aab0d4265481c3361fe188.jpg, createdBy=deb45073082, createdName=14598f55m25暂无昵称, createdTime=Sat Apr 06 08:35:43 CST 2019, time=2019-04-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=361721, encodeId=08e3361e21d8, content=向科研人员致敬！！！, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Thu Feb 28 19:54:14 CST 2019, time=2019-02-28, status=1, ipAttribution=)]
    2019-10-28 751943081_32627960

    #肌营养不良#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1737070, encodeId=7d951e37070cd, content=<a href='/topic/show?id=6ee1810e0ad' target=_blank style='color:#2F92EE;'>#肌营养不良#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81070, encryptionId=6ee1810e0ad, topicName=肌营养不良)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd6034244098, createdName=751943081_32627960, createdTime=Mon Oct 28 23:14:00 CST 2019, time=2019-10-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=368759, encodeId=d5fc368e59fa, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=5b172565856, createdName=12571e9dm53暂无昵称, createdTime=Mon Jul 01 16:08:32 CST 2019, time=2019-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007056, encodeId=68ac200e056b2, content=<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f7482500171, createdName=ms4468361851264128, createdTime=Thu Dec 19 07:14:00 CST 2019, time=2019-12-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=364204, encodeId=cefe36420437, content=这些伟大的科研人员是上帝派来的天使, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cdnapi.center.medsci.cn/medsci/head/2019/04/06/137a837e32aab0d4265481c3361fe188.jpg, createdBy=deb45073082, createdName=14598f55m25暂无昵称, createdTime=Sat Apr 06 08:35:43 CST 2019, time=2019-04-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=361721, encodeId=08e3361e21d8, content=向科研人员致敬！！！, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Thu Feb 28 19:54:14 CST 2019, time=2019-02-28, status=1, ipAttribution=)]
    2019-07-01 12571e9dm53暂无昵称

    厉害

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1737070, encodeId=7d951e37070cd, content=<a href='/topic/show?id=6ee1810e0ad' target=_blank style='color:#2F92EE;'>#肌营养不良#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81070, encryptionId=6ee1810e0ad, topicName=肌营养不良)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd6034244098, createdName=751943081_32627960, createdTime=Mon Oct 28 23:14:00 CST 2019, time=2019-10-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=368759, encodeId=d5fc368e59fa, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=5b172565856, createdName=12571e9dm53暂无昵称, createdTime=Mon Jul 01 16:08:32 CST 2019, time=2019-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007056, encodeId=68ac200e056b2, content=<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f7482500171, createdName=ms4468361851264128, createdTime=Thu Dec 19 07:14:00 CST 2019, time=2019-12-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=364204, encodeId=cefe36420437, content=这些伟大的科研人员是上帝派来的天使, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cdnapi.center.medsci.cn/medsci/head/2019/04/06/137a837e32aab0d4265481c3361fe188.jpg, createdBy=deb45073082, createdName=14598f55m25暂无昵称, createdTime=Sat Apr 06 08:35:43 CST 2019, time=2019-04-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=361721, encodeId=08e3361e21d8, content=向科研人员致敬！！！, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Thu Feb 28 19:54:14 CST 2019, time=2019-02-28, status=1, ipAttribution=)]
    2019-12-19 ms4468361851264128

    #肌营养不良症#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1737070, encodeId=7d951e37070cd, content=<a href='/topic/show?id=6ee1810e0ad' target=_blank style='color:#2F92EE;'>#肌营养不良#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81070, encryptionId=6ee1810e0ad, topicName=肌营养不良)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd6034244098, createdName=751943081_32627960, createdTime=Mon Oct 28 23:14:00 CST 2019, time=2019-10-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=368759, encodeId=d5fc368e59fa, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=5b172565856, createdName=12571e9dm53暂无昵称, createdTime=Mon Jul 01 16:08:32 CST 2019, time=2019-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007056, encodeId=68ac200e056b2, content=<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f7482500171, createdName=ms4468361851264128, createdTime=Thu Dec 19 07:14:00 CST 2019, time=2019-12-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=364204, encodeId=cefe36420437, content=这些伟大的科研人员是上帝派来的天使, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cdnapi.center.medsci.cn/medsci/head/2019/04/06/137a837e32aab0d4265481c3361fe188.jpg, createdBy=deb45073082, createdName=14598f55m25暂无昵称, createdTime=Sat Apr 06 08:35:43 CST 2019, time=2019-04-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=361721, encodeId=08e3361e21d8, content=向科研人员致敬！！！, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Thu Feb 28 19:54:14 CST 2019, time=2019-02-28, status=1, ipAttribution=)]
    2019-04-06 14598f55m25暂无昵称

    这些伟大的科研人员是上帝派来的天使

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1737070, encodeId=7d951e37070cd, content=<a href='/topic/show?id=6ee1810e0ad' target=_blank style='color:#2F92EE;'>#肌营养不良#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81070, encryptionId=6ee1810e0ad, topicName=肌营养不良)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd6034244098, createdName=751943081_32627960, createdTime=Mon Oct 28 23:14:00 CST 2019, time=2019-10-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=368759, encodeId=d5fc368e59fa, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=5b172565856, createdName=12571e9dm53暂无昵称, createdTime=Mon Jul 01 16:08:32 CST 2019, time=2019-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007056, encodeId=68ac200e056b2, content=<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f7482500171, createdName=ms4468361851264128, createdTime=Thu Dec 19 07:14:00 CST 2019, time=2019-12-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=364204, encodeId=cefe36420437, content=这些伟大的科研人员是上帝派来的天使, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cdnapi.center.medsci.cn/medsci/head/2019/04/06/137a837e32aab0d4265481c3361fe188.jpg, createdBy=deb45073082, createdName=14598f55m25暂无昵称, createdTime=Sat Apr 06 08:35:43 CST 2019, time=2019-04-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=361721, encodeId=08e3361e21d8, content=向科研人员致敬！！！, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Thu Feb 28 19:54:14 CST 2019, time=2019-02-28, status=1, ipAttribution=)]
    2019-02-28 坚强007

    向科研人员致敬！！！

    0