Restoration of Functional Full-Length Dystrophin After Intramuscular Transplantation of Foamy Virus-Transduced Myoblasts-Reference-Cited by-同舟云学术

Restoration of Functional Full-Length Dystrophin After Intramuscular Transplantation of Foamy Virus-Transduced Myoblasts

Published:2020-02-01 Issue:3-4 Volume:31 Page:241-252
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Meng Jinhong¹²,Sweeney Nathan Paul³,Doreste Bruno¹²,Muntoni Francesco¹²,McClure Myra³,Morgan Jennifer¹²

Affiliation:

1. Developmental Neuroscience Programme, Molecular Neurosciences Section, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.

2. National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, United Kingdom.

3. Jefferiss Research Trust Laboratories, Imperial College London, London, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.224

Reference67 articles.

1. Evidence-based path to newborn screening for duchenne muscular dystrophy

2. Self-renewal and expansion of single transplanted muscle stem cells

3. Stem Cell Function, Self-Renewal, and Behavioral Heterogeneity of Cells from the Adult Muscle Satellite Cell Niche

4. Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy

5. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

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