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Updated: Jan 21, 2026

A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy
05:54

A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy

Published on: December 29, 2017

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通过修饰基因的上调调节来治疗肌肉衰竭的突变独立方法

Dwi U Kemaladewi1,2, Prabhpreet S Bassi1,3, Steven Erwood1,3

  • 1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Ontario, Canada.

Nature
|July 26, 2019
PubMed
概括

这项研究展示了一种新型的CRISPR激活策略来调节Lama1,这是一种可以修改先天性肌肉衰竭1A型 (MDC1A) 的基因. 这种不依赖突变的方法对治疗MDC1A和其他遗传疾病具有前景.

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Generation of Induced Pluripotent Stem Cells from Muscular Dystrophy Patients: Efficient Integration-free Reprogramming of Urine Derived Cells09:11

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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy09:18

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

Published on: January 12, 2019

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Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
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