Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference45 articles.
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2. Severe adolescent-onset limb-girdle muscular dystrophy due to a novel homozygous nonsense BVES variant;Beecher;J. Neurol. Sci.,2021
3. POPDC1(S201F) causes muscular dystrophy and arrhythmia by affecting protein trafficking;Schindler;J. Clin. Invest.,2016
4. Muscular dystrophy with arrhythmia caused by loss-of-function mutations in BVES;De Ridder;Neurol. Genet.,2019
5. Popeye domain containing proteins are essential for stress-mediated modulation of cardiac pacemaking in mice;Froese;J. Clin. Invest.,2012
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1. Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice;Nature Communications;2024-07-21
2. Systemic Delivery of Full-Length Dystrophin in DMD Mice;2024-05-03
3. Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec;Molecular Therapy - Methods & Clinical Development;2023-09
4. Defective BVES-mediated feedback control of cAMP in muscular dystrophy;Nature Communications;2023-03-30
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