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肌霸登场:肌肉萎缩症的基因疗法靶向 RNA。

Muscling in: Gene therapies for muscular dystrophy target RNA.

机构信息

Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA.

出版信息

Nat Med. 2010 Feb;16(2):170-1. doi: 10.1038/nm0210-170.

Abstract

Muscle diseases can take many forms, from the progressive muscle degeneration of dystrophies to the childhood cancer rhabdomyosarcoma. In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to Bench', Simone Hettmer and Amy J. Wagers examine the implications of clinical studies describing a type of rhabdomyosarcoma that resembles acute leukemia. The findings dovetail with other studies suggesting that some of these cancers might originate outside of muscle tissue and highlight the need for a better understanding of the cells that give rise to this condition.

摘要

肌肉疾病有多种形式,从营养不良导致的进行性肌肉退化到儿童癌症横纹肌肉瘤。在《从实验室到临床》一书中,乔尔·R·张伯伦(Joel R. Chamberlain)和杰弗里·S·张伯伦(Jeffrey S. Chamberlain)讨论了使用反义寡核苷酸治疗杜氏肌营养不良症和强直性肌营养不良症的研究。在《从临床到实验室》一书中,西蒙妮·赫特默(Simone Hettmer)和艾米·J·瓦格斯(Amy J. Wagers)探讨了描述一种类似于急性白血病的横纹肌肉瘤的临床研究的意义。这些发现与其他研究相吻合,表明这些癌症中的一些可能起源于肌肉组织之外,并强调需要更好地了解导致这种疾病的细胞。

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