Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion

In a previous limb-girdle muscular dystrophy type 2D (LGMD2D) clinical trial, robust alpha-sarcoglycan gene expression was confirmed following intramuscular gene (SGCA) transfer. This paved the way for first-in-human isolated limb infusion (ILI) gene transfer trial to the lower limbs. Delivery of sc...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Mendell, Jerry R., Chicoine, Louis G., Al-Zaidy, Samiah A., Sahenk, Zarife, Lehman, Kelly, Lowes, Linda, Miller, Natalie, Alfano, Lindsay, Galliers, Beverly, Lewis, Sarah, Murrey, Darren, Peterson, Ellyn, Griffin, Danielle A., Church, Kathleen, Cheatham, Sharon, Cheatham, John, Hogan, Mark J., Rodino-Klapac, Louise R.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2019
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6648191/
https://ncbi.nlm.nih.gov/pubmed/30838895
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.006
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