Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy

Limb-girdle muscular dystrophy type 2D/R3 (LGMD2D/R3) is a progressive muscular dystrophy that manifests with muscle weakness, respiratory abnormalities, and in rare cases cardiomyopathy. LGMD2D/R3 is caused by mutations in the SGCA gene resulting in loss of protein and concomitant loss of some or a...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
में प्रकाशित:Hum Gene Ther
मुख्य लेखकों: Griffin, Danielle A., Pozsgai, Eric R., Heller, Kristin N., Potter, Rachael A., Peterson, Ellyn L., Rodino-Klapac, Louise R.
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: Mary Ann Liebert, Inc., publishers 2021
विषय:
Research Articles
ऑनलाइन पहुंच:https://ncbi.nlm.nih.gov/pmc/articles/PMC8066346/
https://ncbi.nlm.nih.gov/pubmed/33349138
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.199
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