肌萎缩症的原理与实践

English | 2024 | ISBN: 3031440080 | 362 Pages | PDF EPUB (True) | 130 MB

The field of muscular dystrophies has expanded significantly with the discovery of the genetic defects and protein products underlying these disorders. New treatments such as antisense oligonucleotides and siRNAs and gene therapies are now in clinical application and in trials. As clinical trials increase, outcome measurement becomes very important, and more validated tools are being developed.

肌肉 dystrophies 的领域随着对这些疾病背后的遗传缺陷和蛋白质产品发现而显著扩大。目前，新的治疗方法如 antisense oligonucleotides 和 siRNAs 以及基因疗法现在已进入临床应用和试验阶段。随着临床试验的增加，结果测量变得非常重要，越来越多验证过的工具也在开发中。

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