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亨廷顿病样 2 型进行性骨化性纤维发育不良治疗的研究进展与趋势。

Progress and trends in the development of therapies for Hutchinson-Gilford progeria syndrome.

机构信息

School of Life and Health Sciences, The Chinese University of Hong Kong (Shenzhen), Shenzhen, China.

Department of Applied Biology and Chemical Technology, Hong Kong Polytechnic University, Hong Kong Special Administrative Region, China.

出版信息

Aging Cell. 2020 Jul;19(7):e13175. doi: 10.1111/acel.13175. Epub 2020 Jun 28.

DOI:10.1111/acel.13175
PMID:32596971
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7370734/
Abstract

Hutchinson-Gilford progeria syndrome (HGPS) is an autosomal-dominant genetic disease that leads to accelerated aging and often premature death caused by cardiovascular complications. Till now clinical management of HGPS has largely relied on the treatment of manifestations and on the prevention of secondary complications, cure for the disease has not yet been established. Addressing this need cannot only benefit progeria patients but may also provide insights into intervention design for combating physiological aging. By using the systematic review approach, this article revisits the overall progress in the development of strategies for HGPS treatment over the last ten years, from 2010 to 2019. In total, 1,906 articles have been retrieved, of which 56 studies have been included for further analysis. Based on the articles analyzed, the trends in the use of different HGPS models, along with the prevalence, efficiency, and limitations of different reported treatment strategies, have been examined. Emerging strategies for preclinical studies, and possible targets for intervention development, have also been presented as avenues for future research.

摘要

亨廷顿舞蹈症-吉福德早衰综合征(HGPS)是一种常染色体显性遗传疾病,可导致心血管并发症,从而加速衰老和过早死亡。到目前为止,HGPS 的临床管理主要依赖于对临床表现的治疗和对继发性并发症的预防,尚未建立针对该疾病的治愈方法。满足这一需求不仅可以使早衰症患者受益,而且还可能为干预设计提供对抗生理性衰老的见解。本文通过系统回顾的方法,回顾了 2010 年至 2019 年过去十年中 HGPS 治疗策略的整体进展。共检索到 1906 篇文章,其中有 56 项研究被纳入进一步分析。根据分析的文章,检查了不同 HGPS 模型的使用趋势,以及不同报道的治疗策略的普遍性、效率和局限性。还提出了用于临床前研究的新兴策略,以及干预开发的可能目标,作为未来研究的途径。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3f44/7370734/0d6a5ced5ff8/ACEL-19-e13175-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3f44/7370734/0d6a5ced5ff8/ACEL-19-e13175-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3f44/7370734/0d6a5ced5ff8/ACEL-19-e13175-g001.jpg

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Nat Commun. 2019 Nov 18;10(1):4990. doi: 10.1038/s41467-019-13018-3.
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