Baidu
map

里程碑研究显示,天然分子尿石素A能改善肌营养不良症小鼠的肌肉功能和预期寿命

2021-04-12 国际文传 国际文传

《科学转化医学》发表瑞士科学家的研究成果,显示线粒体吞噬诱导剂尿石素A能恢复杜氏肌营养不良症模型中的肌细胞活性

《科学转化医学》发表瑞士科学家的研究成果,显示线粒体吞噬诱导剂尿石素A能恢复杜氏肌营养不良症模型中的肌细胞活性

今天报道的新结果显示,小鼠饮食中补充尿石素A能延缓其杜氏肌营养不良症(DMD)的进展。上述结果发表于《科学转化医学》(Science Translational Medicine),有朝一日有望实现为DMD开发新的治疗方案,DMD是一种无法治愈的基因疾病,其特征是进行性肌肉退化。每3,500名新生男孩中约有1例患DMD,通常在儿童期发病,大幅缩短预期寿命。

此项新研究在瑞士洛桑联邦理工学院(EPFL)和洛桑大学教授Johan Auwerx, MD博士的实验室开展,合作方是瑞士生命科学公司Amazentis的科学家们,研究强调,线粒体缺陷能在DMD中发挥重要作用。线粒体是细胞的动力源,产生正常肌肉功能所需的能量。但该研究发现,从人类DMD患者和为模拟DMD而繁殖的小鼠中提取的肌细胞,均显示有线粒体活性的显著缺陷。具体而言,基因表达模式显示,DMD的发病与线粒体吞噬的明显减少有关,细胞依赖线粒体吞噬这一过程来清除和回收有缺陷的线粒体、维持高能量水平。

主要作者、EPFL教授Johan Auwerx, MD博士表示:“杜氏肌营养不良症是儿童期最常见的致命基因疾病,至今仍无治愈手段。我们的研究是寻找肌营养不良症新治疗方法中的重大突破。

已知天然化合物尿石素A可激活小鼠和人类线粒体吞噬,改善线粒体健康。研究科学家、主要作者Peiling Luan和Davide D'Amico给DMD小鼠喂食尿石素A仅10周时,就发现线粒体吞噬水平有效升高,恢复至正常。肌肉损伤由此明显减少,肌肉健康和性能改善。DMD小鼠中,与未处理的对照组相比,尿石素A施用组的抓地力高31%,奔跑能力高45%,寿命更长,存活率高40%

尿石素A将DMD小鼠心脏和横膈膜肌中称为纤维化的破坏性疾病分别减少36%39%,这对于人类的DMD至为重要。DMD患者中所见的类似病损通常可引起致死性心脏或呼吸衰竭。尿石素同时能增强小鼠肌肉干细胞再生。这对人类DMD疾病特别有现实意义,因为DMD的发作与功能性干细胞的衰竭有关。

Amazentis项目主管、该论文第一作者Davide D’Amico博士表示:“此项研究之前,已了解到DMD患者肌肉功能的急剧丧失与线粒体功能障碍有关。在这里,我们发现线粒体吞噬(失能线粒体的清除和回收)的缺陷在DMD进展中发挥关键作用。

Amazentis联合创始人兼首席执行官Chris Rinsch博士表示:“《科学转化医学》发表的严谨科学结果进一步证实尿石素A作为肌肉功能的强效增强剂的科学证据。令人振奋的是,这种天然代谢产物不仅能支持健康的肌肉,而且有临床前研究显示其有望治疗进行性肌病。”

当期《科学转化医学》上题为“尿石素A可通过挽救肌营养不良症的线粒体吞噬来改善肌肉功能”的论文报道了上述结果。

尿石素A的背景

Amazentis的专有先导产品是尿石素A的口服制剂。尿石素A是微生物来源的鞣花单宁的代谢产物,鞣花单宁是在石榴及其他水果和坚果中发现的一类化合物。施用尿石素A可通过刺激线粒体吞噬来改善线粒体功能,从而刺激健康线粒体的生长,线粒体吞噬是从细胞中清除衰老和受损线粒体的过程。细胞内的线粒体吞噬随年龄增长而减少,而肌肉中线粒体功能减退被认为是年龄相关肌肉衰弱的主要原因之一。此前,Amazentis与EPFL在《自然杂志·医学分册》上题为“尿石素A可诱导秀丽隐杆线虫线粒体吞噬,延长其寿命,增强啮齿类肌肉功能”的论文(doi:10.1038/nm.4132)中报道了临床前结果,并在《自然杂志·代谢分册》上题为“线粒体吞噬激活剂尿石素A是安全的,可诱导人类线粒体和细胞健康改善的分子特征”的论文(doi: 10.1038/s42255-019-0073-4)中报道了临床结果,展示了尿石素A的安全性、生物利用度及其对线粒体健康生物标志物的影响。

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 (7)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=2160833, encodeId=37c72160833a5, content=<a href='/topic/show?id=98984e48182' target=_blank style='color:#2F92EE;'>#尿石素A#</a>能改善<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>小鼠的<a href='/topic/show?id=a85180981ee' target=_blank style='color:#2F92EE;'>#肌肉功能#</a>和预期寿命, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47481, encryptionId=98984e48182, topicName=尿石素A), TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症), TopicDto(id=80981, encryptionId=a85180981ee, topicName=肌肉功能)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/Random/55971dc507c93968175ce7cc1e177b372a83869f.jpg, createdBy=f63e4754896, createdName=侠胆医心, createdTime=Wed Oct 04 06:31:34 CST 2023, time=2023-10-04, status=1, ipAttribution=上海), GetPortalCommentsPageByObjectIdResponse(id=1737088, encodeId=7d361e3708853, 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=92, 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=Tue Nov 16 08:16:38 CST 2021, time=2021-11-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007069, encodeId=c065200e0691c, 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=88, 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 Jun 17 03:16:38 CST 2021, time=2021-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2059327, encodeId=f631205932e38, content=<a href='/topic/show?id=9fbe95e0015' target=_blank style='color:#2F92EE;'>#里程碑#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95700, encryptionId=9fbe95e0015, topicName=里程碑)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Wed Jun 16 04:16:38 CST 2021, time=2021-06-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=957003, encodeId=56ba95e003e3, content=<a href='/topic/show?id=f5174119e20' target=_blank style='color:#2F92EE;'>#图片#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=1, topicName=null, topicId=null, topicList=[TopicDto(id=41197, encryptionId=f5174119e20, topicName=图片)], attachment=[AttachmentDto(url=https://img.medsci.cn/comment/20210414/f01ec18818814b4bbe0cc51a6d4ca3bf.jpeg, type=image, width=780, height=658)], authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210706/5924e86cc73c43efb5bad9dd92ac0d7b/63609a5527c447f48b59b39dd248a66f.jpg, createdBy=cf0a5102349, createdName=站在塔尖, createdTime=Wed Apr 14 18:19:18 CST 2021, time=2021-04-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1315882, encodeId=b3f01315882dd, content=<a href='/topic/show?id=bbc8100216a0' target=_blank style='color:#2F92EE;'>#预期寿命#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=100216, encryptionId=bbc8100216a0, topicName=预期寿命)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ba58436, createdName=马龙, createdTime=Wed Apr 14 08:16:38 CST 2021, time=2021-04-14, status=1, ipAttribution=)]
    2023-10-04 侠胆医心 来自上海

    #尿石素A#能改善#肌营养不良症#小鼠的#肌肉功能#和预期寿命

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=2160833, encodeId=37c72160833a5, content=<a href='/topic/show?id=98984e48182' target=_blank style='color:#2F92EE;'>#尿石素A#</a>能改善<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>小鼠的<a href='/topic/show?id=a85180981ee' target=_blank style='color:#2F92EE;'>#肌肉功能#</a>和预期寿命, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47481, encryptionId=98984e48182, topicName=尿石素A), TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症), TopicDto(id=80981, encryptionId=a85180981ee, topicName=肌肉功能)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/Random/55971dc507c93968175ce7cc1e177b372a83869f.jpg, createdBy=f63e4754896, createdName=侠胆医心, createdTime=Wed Oct 04 06:31:34 CST 2023, time=2023-10-04, status=1, ipAttribution=上海), GetPortalCommentsPageByObjectIdResponse(id=1737088, encodeId=7d361e3708853, 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=92, 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=Tue Nov 16 08:16:38 CST 2021, time=2021-11-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007069, encodeId=c065200e0691c, 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=88, 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 Jun 17 03:16:38 CST 2021, time=2021-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2059327, encodeId=f631205932e38, content=<a href='/topic/show?id=9fbe95e0015' target=_blank style='color:#2F92EE;'>#里程碑#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95700, encryptionId=9fbe95e0015, topicName=里程碑)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Wed Jun 16 04:16:38 CST 2021, time=2021-06-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=957003, encodeId=56ba95e003e3, content=<a href='/topic/show?id=f5174119e20' target=_blank style='color:#2F92EE;'>#图片#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=1, topicName=null, topicId=null, topicList=[TopicDto(id=41197, encryptionId=f5174119e20, topicName=图片)], attachment=[AttachmentDto(url=https://img.medsci.cn/comment/20210414/f01ec18818814b4bbe0cc51a6d4ca3bf.jpeg, type=image, width=780, height=658)], authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210706/5924e86cc73c43efb5bad9dd92ac0d7b/63609a5527c447f48b59b39dd248a66f.jpg, createdBy=cf0a5102349, createdName=站在塔尖, createdTime=Wed Apr 14 18:19:18 CST 2021, time=2021-04-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1315882, encodeId=b3f01315882dd, content=<a href='/topic/show?id=bbc8100216a0' target=_blank style='color:#2F92EE;'>#预期寿命#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=100216, encryptionId=bbc8100216a0, topicName=预期寿命)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ba58436, createdName=马龙, createdTime=Wed Apr 14 08:16:38 CST 2021, time=2021-04-14, status=1, ipAttribution=)]
  3. [GetPortalCommentsPageByObjectIdResponse(id=2160833, encodeId=37c72160833a5, content=<a href='/topic/show?id=98984e48182' target=_blank style='color:#2F92EE;'>#尿石素A#</a>能改善<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>小鼠的<a href='/topic/show?id=a85180981ee' target=_blank style='color:#2F92EE;'>#肌肉功能#</a>和预期寿命, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47481, encryptionId=98984e48182, topicName=尿石素A), TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症), TopicDto(id=80981, encryptionId=a85180981ee, topicName=肌肉功能)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/Random/55971dc507c93968175ce7cc1e177b372a83869f.jpg, createdBy=f63e4754896, createdName=侠胆医心, createdTime=Wed Oct 04 06:31:34 CST 2023, time=2023-10-04, status=1, ipAttribution=上海), GetPortalCommentsPageByObjectIdResponse(id=1737088, encodeId=7d361e3708853, 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=92, 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=Tue Nov 16 08:16:38 CST 2021, time=2021-11-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007069, encodeId=c065200e0691c, 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=88, 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 Jun 17 03:16:38 CST 2021, time=2021-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2059327, encodeId=f631205932e38, content=<a href='/topic/show?id=9fbe95e0015' target=_blank style='color:#2F92EE;'>#里程碑#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95700, encryptionId=9fbe95e0015, topicName=里程碑)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Wed Jun 16 04:16:38 CST 2021, time=2021-06-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=957003, encodeId=56ba95e003e3, content=<a href='/topic/show?id=f5174119e20' target=_blank style='color:#2F92EE;'>#图片#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=1, topicName=null, topicId=null, topicList=[TopicDto(id=41197, encryptionId=f5174119e20, topicName=图片)], attachment=[AttachmentDto(url=https://img.medsci.cn/comment/20210414/f01ec18818814b4bbe0cc51a6d4ca3bf.jpeg, type=image, width=780, height=658)], authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210706/5924e86cc73c43efb5bad9dd92ac0d7b/63609a5527c447f48b59b39dd248a66f.jpg, createdBy=cf0a5102349, createdName=站在塔尖, createdTime=Wed Apr 14 18:19:18 CST 2021, time=2021-04-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1315882, encodeId=b3f01315882dd, content=<a href='/topic/show?id=bbc8100216a0' target=_blank style='color:#2F92EE;'>#预期寿命#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=100216, encryptionId=bbc8100216a0, topicName=预期寿命)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ba58436, createdName=马龙, createdTime=Wed Apr 14 08:16:38 CST 2021, time=2021-04-14, status=1, ipAttribution=)]
  4. [GetPortalCommentsPageByObjectIdResponse(id=2160833, encodeId=37c72160833a5, content=<a href='/topic/show?id=98984e48182' target=_blank style='color:#2F92EE;'>#尿石素A#</a>能改善<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>小鼠的<a href='/topic/show?id=a85180981ee' target=_blank style='color:#2F92EE;'>#肌肉功能#</a>和预期寿命, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47481, encryptionId=98984e48182, topicName=尿石素A), TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症), TopicDto(id=80981, encryptionId=a85180981ee, topicName=肌肉功能)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/Random/55971dc507c93968175ce7cc1e177b372a83869f.jpg, createdBy=f63e4754896, createdName=侠胆医心, createdTime=Wed Oct 04 06:31:34 CST 2023, time=2023-10-04, status=1, ipAttribution=上海), GetPortalCommentsPageByObjectIdResponse(id=1737088, encodeId=7d361e3708853, 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=92, 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=Tue Nov 16 08:16:38 CST 2021, time=2021-11-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007069, encodeId=c065200e0691c, 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=88, 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 Jun 17 03:16:38 CST 2021, time=2021-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2059327, encodeId=f631205932e38, content=<a href='/topic/show?id=9fbe95e0015' target=_blank style='color:#2F92EE;'>#里程碑#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95700, encryptionId=9fbe95e0015, topicName=里程碑)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Wed Jun 16 04:16:38 CST 2021, time=2021-06-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=957003, encodeId=56ba95e003e3, content=<a href='/topic/show?id=f5174119e20' target=_blank style='color:#2F92EE;'>#图片#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=1, topicName=null, topicId=null, topicList=[TopicDto(id=41197, encryptionId=f5174119e20, topicName=图片)], attachment=[AttachmentDto(url=https://img.medsci.cn/comment/20210414/f01ec18818814b4bbe0cc51a6d4ca3bf.jpeg, type=image, width=780, height=658)], authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210706/5924e86cc73c43efb5bad9dd92ac0d7b/63609a5527c447f48b59b39dd248a66f.jpg, createdBy=cf0a5102349, createdName=站在塔尖, createdTime=Wed Apr 14 18:19:18 CST 2021, time=2021-04-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1315882, encodeId=b3f01315882dd, content=<a href='/topic/show?id=bbc8100216a0' target=_blank style='color:#2F92EE;'>#预期寿命#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=100216, encryptionId=bbc8100216a0, topicName=预期寿命)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ba58436, createdName=马龙, createdTime=Wed Apr 14 08:16:38 CST 2021, time=2021-04-14, status=1, ipAttribution=)]
    2021-06-16 xzw113
  5. [GetPortalCommentsPageByObjectIdResponse(id=2160833, encodeId=37c72160833a5, content=<a href='/topic/show?id=98984e48182' target=_blank style='color:#2F92EE;'>#尿石素A#</a>能改善<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>小鼠的<a href='/topic/show?id=a85180981ee' target=_blank style='color:#2F92EE;'>#肌肉功能#</a>和预期寿命, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47481, encryptionId=98984e48182, topicName=尿石素A), TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症), TopicDto(id=80981, encryptionId=a85180981ee, topicName=肌肉功能)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/Random/55971dc507c93968175ce7cc1e177b372a83869f.jpg, createdBy=f63e4754896, createdName=侠胆医心, createdTime=Wed Oct 04 06:31:34 CST 2023, time=2023-10-04, status=1, ipAttribution=上海), GetPortalCommentsPageByObjectIdResponse(id=1737088, encodeId=7d361e3708853, 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=92, 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=Tue Nov 16 08:16:38 CST 2021, time=2021-11-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007069, encodeId=c065200e0691c, 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=88, 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 Jun 17 03:16:38 CST 2021, time=2021-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2059327, encodeId=f631205932e38, content=<a href='/topic/show?id=9fbe95e0015' target=_blank style='color:#2F92EE;'>#里程碑#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95700, encryptionId=9fbe95e0015, topicName=里程碑)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Wed Jun 16 04:16:38 CST 2021, time=2021-06-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=957003, encodeId=56ba95e003e3, content=<a href='/topic/show?id=f5174119e20' target=_blank style='color:#2F92EE;'>#图片#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=1, topicName=null, topicId=null, topicList=[TopicDto(id=41197, encryptionId=f5174119e20, topicName=图片)], attachment=[AttachmentDto(url=https://img.medsci.cn/comment/20210414/f01ec18818814b4bbe0cc51a6d4ca3bf.jpeg, type=image, width=780, height=658)], authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210706/5924e86cc73c43efb5bad9dd92ac0d7b/63609a5527c447f48b59b39dd248a66f.jpg, createdBy=cf0a5102349, createdName=站在塔尖, createdTime=Wed Apr 14 18:19:18 CST 2021, time=2021-04-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1315882, encodeId=b3f01315882dd, content=<a href='/topic/show?id=bbc8100216a0' target=_blank style='color:#2F92EE;'>#预期寿命#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=100216, encryptionId=bbc8100216a0, topicName=预期寿命)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ba58436, createdName=马龙, createdTime=Wed Apr 14 08:16:38 CST 2021, time=2021-04-14, status=1, ipAttribution=)]
  6. [GetPortalCommentsPageByObjectIdResponse(id=2160833, encodeId=37c72160833a5, content=<a href='/topic/show?id=98984e48182' target=_blank style='color:#2F92EE;'>#尿石素A#</a>能改善<a href='/topic/show?id=21fe810e29d' target=_blank style='color:#2F92EE;'>#肌营养不良症#</a>小鼠的<a href='/topic/show?id=a85180981ee' target=_blank style='color:#2F92EE;'>#肌肉功能#</a>和预期寿命, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47481, encryptionId=98984e48182, topicName=尿石素A), TopicDto(id=81072, encryptionId=21fe810e29d, topicName=肌营养不良症), TopicDto(id=80981, encryptionId=a85180981ee, topicName=肌肉功能)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/Random/55971dc507c93968175ce7cc1e177b372a83869f.jpg, createdBy=f63e4754896, createdName=侠胆医心, createdTime=Wed Oct 04 06:31:34 CST 2023, time=2023-10-04, status=1, ipAttribution=上海), GetPortalCommentsPageByObjectIdResponse(id=1737088, encodeId=7d361e3708853, 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=92, 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=Tue Nov 16 08:16:38 CST 2021, time=2021-11-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2007069, encodeId=c065200e0691c, 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=88, 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 Jun 17 03:16:38 CST 2021, time=2021-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2059327, encodeId=f631205932e38, content=<a href='/topic/show?id=9fbe95e0015' target=_blank style='color:#2F92EE;'>#里程碑#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95700, encryptionId=9fbe95e0015, topicName=里程碑)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Wed Jun 16 04:16:38 CST 2021, time=2021-06-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=957003, encodeId=56ba95e003e3, content=<a href='/topic/show?id=f5174119e20' target=_blank style='color:#2F92EE;'>#图片#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=1, topicName=null, topicId=null, topicList=[TopicDto(id=41197, encryptionId=f5174119e20, topicName=图片)], attachment=[AttachmentDto(url=https://img.medsci.cn/comment/20210414/f01ec18818814b4bbe0cc51a6d4ca3bf.jpeg, type=image, width=780, height=658)], authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210706/5924e86cc73c43efb5bad9dd92ac0d7b/63609a5527c447f48b59b39dd248a66f.jpg, createdBy=cf0a5102349, createdName=站在塔尖, createdTime=Wed Apr 14 18:19:18 CST 2021, time=2021-04-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1315882, encodeId=b3f01315882dd, content=<a href='/topic/show?id=bbc8100216a0' target=_blank style='color:#2F92EE;'>#预期寿命#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=100216, encryptionId=bbc8100216a0, topicName=预期寿命)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ba58436, createdName=马龙, createdTime=Wed Apr 14 08:16:38 CST 2021, time=2021-04-14, status=1, ipAttribution=)]

拓展阅读

Nature Medicine:杜氏肌营养不良症的AAV基因疗法

尽管delandistrogene moxeparvovec在主要终点NSAA评分变化上未能显示出统计学显著优势,但在微抗肌萎缩蛋白表达和其他次要终点上显示出一定的积极趋势。

杜氏肌营养不良症发生心脏病的疾病预测因子研究

通过此次系统文献回顾,对DMD心脏疾病的预测因子有了更深入的了解。未来的研究应继续探索这些预测因子的具体机制,以便更好地指导临床实践,提高DMD患者的护理质量。

《柳叶刀-区域健康》:中国Duchenne型肌营养不良症患者疾病自然史研究的1年分析

DMD疾病进展到临床里程碑的时间与既往研究发现一致,其中行走能力丧失发生在13岁。

一项关于他莫昔芬治疗杜氏肌营养不良症安全性和有效性的临床研究

DMD男性患儿使用他莫昔芬治疗是安全且耐受性良好的,与安慰剂组相比,他莫昔芬治疗组的疾病进展较慢(定义为运动功能随时间的丧失),但结果指标的差异既无临床意义也无统计学意义。

首份同类临床报告显示,日本的Neu-REFIX® Beta 1,3-1,6葡聚糖*有望改变杜氏肌营养不良症的病情

杜氏肌营养不良症(DMD),这是一种罕见的遗传性疾病,日本约有5000名患者,海湾合作委员会约有3000名患者,美国有不到5万名患者。

杜氏肌营养不良症基因疗法FDA听证会实录,历史首次!

FDA于2023年5月12日召集外部专家会议,审查Sarepta Therapeutics公司针对杜氏肌营养不良症(DMD)的实验性基因疗法的临床数据。这场为期一整天的会议事关重大。STAT跟踪报道了

2023 NICE 高度专业化的技术指导:阿塔鲁伦用于治疗抗肌萎缩蛋白基因无义突变的杜氏肌营养不良症【HST22】

英国国家卫生与临床优化研究所(NICE,National Institute for Health and Clinical Excellence) · 2023-02-22

Baidu
map
Baidu
map
Baidu
map