Systemic AAV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases

Estrogen has been implicated in muscle membrane stability,6 and testosterone has a well-known influence on muscle fiber size.7 Such differences might translate into functional differences between the treated and control groups. [...]the reader might wish to focus on the comparisons between the diffe...

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Bibliographic Details
Published in:Molecular therapy 2017-04, Vol.25 (4), p.834-835
Main Author: Wells, Dominic J.
Format: Article
Language:English
Subjects:
Animal models
Atrophy
Becker's muscular dystrophy
Clinical trials
Dependovirus - genetics
Disease
Duchenne's muscular dystrophy
Dystrophin
Follistatin
Follistatin gene
Gene therapy
Genetic Therapy
Genetic Vectors
Humans
Muscular dystrophy
Myositis
Neuromuscular Diseases
Patients
Spinal muscular atrophy
Statistical analysis
Studies
Testosterone
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Summary:Estrogen has been implicated in muscle membrane stability,6 and testosterone has a well-known influence on muscle fiber size.7 Such differences might translate into functional differences between the treated and control groups. [...]the reader might wish to focus on the comparisons between the different treatment groups, which show a clear dose response for many of the outcome measures performed with the dogs. [...]the dramatic amelioration of the disease seen in the murine and canine models of XLMTM may not be precisely replicated by a similar improvement in man. Clinicaltrials.gov Identifier Trial Route Associated Publications NCT00428935 safety study of mini-dystrophin gene to treat Duchenne muscular dystrophy intramuscular Mendell et al.11 NCT00494195 gene transfer therapy for treating children and adults with limb girdle muscular dystrophy type 2D (LGMD2D) intramuscular Mendell et al.12 NCT01344798 clinical study of AAV1-gamma-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C intramuscular none NCT01519349 follistatin gene transfer to patients with Becker muscular dystrophy and sporadic inclusion body myositis intramuscular Mendell et al.13 and Al-Zaidy14 NCT02122952 gene transfer clinical trial for spinal muscular atrophy type 1 systemic (IV) ongoing NCT02354781 clinical intramuscular gene transfer of rAAV1.CMV.huFollistatin344 trial to patients with Duchenne muscular dystrophy intramuscular ongoing Table 1 A Summary of Human Clinical Trials of AAV-Based Gene Transfer in Neuromuscular Diseases
ISSN:1525-0016
1525-0024
DOI:10.1016/j.ymthe.2017.03.006