Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

A recombinant serotype 9 adeno-associated virus (rAAV9) vector carrying a transgene that expresses codon-optimized human acid alpha-glucosidase (hGAA, or GAA) driven by a human desmin (DES) promoter (i.e., rAAV9-DES-hGAA) has been generated as a clinical candidate vector for Pompe disease. The rAAV9...

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Publicat a:Hum Gene Ther Clin Dev
Autors principals: Corti, Manuela, Cleaver, Brian, Clément, Nathalie, Conlon, Thomas J., Faris, Kaitlyn J., Wang, Gensheng, Benson, Janet, Tarantal, Alice F., Fuller, Davis, Herzog, Roland W., Byrne, Barry J.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2015
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4606909/
https://ncbi.nlm.nih.gov/pubmed/26390092
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.068
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