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

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Progress and trends in the development of therapies for Hutchinson-Gilford progeria syndrome.
Aging Cell ( IF 8.0 ) Pub Date : 2020-06-28 , DOI: 10.1111/acel.13175
Wing-Fu Lai _{1,

2} , Wing-Tak Wong ₂

Affiliation

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.

中文翻译：

Hutchinson-Gilford早衰综合症治疗方法的进展和趋势。

Hutchinson–Gilford早衰综合症（HGPS）是常染色体显性遗传病，可导致衰老加速，并经常因心血管并发症而过早死亡。到目前为止，HGPS的临床管理在很大程度上依赖于表现的治疗以及对继发并发症的预防，目前尚无治愈该病的方法。解决这一需求不仅可以使早衰患者受益，还可以为对抗生理衰老的干预设计提供见识。通过使用系统评价方法，本文回顾了从2010年至2019年的过去十年中HGPS治疗策略发展的总体进展。总共检索到1,906篇文章，其中包括56篇研究以进一步研究分析。根据分析的文章，研究了不同HGPS模型的使用趋势，以及不同报道的治疗策略的普遍性，有效性和局限性。临床前研究的新兴策略以及干预发展的可能目标也已被提出作为未来研究的途径。

更新日期：2020-06-28

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