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Hum Mol Genet:利用蠕虫成功筛选出致命大脑疾病的候选药物

2014-07-21 佚名 生物谷

近日,利物浦大学的研究人员已经成功建造出一种无法治愈的、致命神经退行性疾病的蠕虫模型,并利用此蠕虫模型,筛选到一种能治疗疾病的潜在化合物。 在欧洲和北美,10万中就有1人会患神经元蜡样质脂褐质沉积症 (ANCL),ANCL通常在30多岁发病,在40多岁左右导致死亡。虽然最近鉴定出此疾病是由DNAJC5基因被突变引起的,目前还没有已知的手段来治疗这种疾病。 这是第一次科学家在蠕虫中成功复制该疾病,

近日,利物浦大学的研究人员已经成功建造出一种无法治愈的、致命神经退行性疾病的蠕虫模型,并利用此蠕虫模型,筛选到一种能治疗疾病的潜在化合物。

在欧洲和北美,10万中就有1人会患神经元蜡样质脂褐质沉积症 (ANCL),ANCL通常在30多岁发病,在40多岁左右导致死亡。虽然最近鉴定出此疾病是由DNAJC5基因被突变引起的,目前还没有已知的手段来治疗这种疾病。

这是第一次科学家在蠕虫中成功复制该疾病,并成功用一个化合物白藜芦醇来治疗该疾病。由于该蠕虫的寿命通常只有几个星期,因此疾病症状会在几天内显现,这就打开了在短期内测试用于治疗疾病的数以千计新化合物的可能性。

一种化合物白藜芦醇已经由研究小组证明能有效地治疗疾病,并是以未知的方式来发挥作用。生理学家Alan Morgan教授领导了这项研究,他说:ANCL是罕见的,目前它是无法治愈的。这项研究使我们能够快速测试可用于治疗的化合物,在利用我们的模型所测试的第一批化合物中,其中一个(白藜芦醇)已经显示出令人鼓舞的效果。

Morgan教授和他的同事Bob Burgoyne教授、博士研究生Sudhanva Kashyap现在计划利用蠕虫模型测试更多的化合物,并探讨蠕虫如何被用于研究多种神经退化性疾病。

原始出处:


Kashyap SS1, Johnson JR1, McCue HV1, Chen X1, Edmonds MJ1, Ayala M1, Graham ME1, Jenn RC1, Barclay JW1, Burgoyne RD1, Morgan A2.Caenorhabditis elegans dnj-14, the orthologue of the DNAJC5 gene mutated in adult onset neuronal ceroid lipofuscinosis, provides a new platform for neuroprotective drug screening and identifies a SIR-2.1-independent action of resveratrol.Hum Mol Genet. 2014 Jun 19. pii: ddu316. [pdf free]

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    2014-09-17 cy0324
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  6. [GetPortalCommentsPageByObjectIdResponse(id=1685956, encodeId=382116859561d, content=<a href='/topic/show?id=66f3e93862' target=_blank style='color:#2F92EE;'>#Genet#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=65, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7938, encryptionId=66f3e93862, topicName=Genet)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d3e128296421, createdName=江川靖瑶, createdTime=Sat Oct 04 08:47:00 CST 2014, time=2014-10-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2052394, encodeId=cd262052394ec, content=<a href='/topic/show?id=688a2e469f0' target=_blank style='color:#2F92EE;'>#候选药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=76, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=27469, encryptionId=688a2e469f0, topicName=候选药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=467c360, createdName=yuanming7, createdTime=Thu Dec 04 15:47:00 CST 2014, time=2014-12-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2009616, encodeId=ef6a20096161a, content=<a href='/topic/show?id=2fc84393499' target=_blank style='color:#2F92EE;'>#大脑疾病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=99, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=43934, encryptionId=2fc84393499, topicName=大脑疾病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8dba135, createdName=xuyong523, createdTime=Mon Apr 13 08:47:00 CST 2015, time=2015-04-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2048605, encodeId=579f2048605d2, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Wed Sep 17 20:47:00 CST 2014, time=2014-09-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1672119, encodeId=8f8316e2119a6, content=<a href='/topic/show?id=adfd8494496' target=_blank style='color:#2F92EE;'>#脑疾病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84944, encryptionId=adfd8494496, topicName=脑疾病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=62f626885922, createdName=chendoc251, createdTime=Fri Mar 20 07:47:00 CST 2015, time=2015-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1733701, encodeId=46981e3370101, content=<a href='/topic/show?id=d4f412668a4' target=_blank style='color:#2F92EE;'>#NET#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=48, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=12668, encryptionId=d4f412668a4, topicName=NET)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7ea033781210, createdName=canlab, createdTime=Fri Apr 10 15:47:00 CST 2015, time=2015-04-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1694358, encodeId=cf6d1694358a5, content=<a href='/topic/show?id=12688890867' target=_blank style='color:#2F92EE;'>#蠕虫#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88908, encryptionId=12688890867, topicName=蠕虫)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=20d629606482, createdName=smlt2018, createdTime=Fri Jun 05 23:47:00 CST 2015, time=2015-06-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1584334, encodeId=9f7a158433432, content=<a href='/topic/show?id=9bbee5948f0' target=_blank style='color:#2F92EE;'>#筛选#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=75948, encryptionId=9bbee5948f0, topicName=筛选)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=39da17047364, createdName=ms6747015003810650, createdTime=Wed Jul 23 00:47:00 CST 2014, time=2014-07-23, status=1, ipAttribution=)]
    2015-04-10 canlab
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    2015-06-05 smlt2018
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