Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice.
Publication/Presentation Date
10-25-2024
Abstract
Mucopolysaccharidosis type IVA (MPS IVA) is an autosomal congenital metabolic lysosomal disease caused by a deficiency of the
ISSN
1557-7422
Published In/Presented At
Herreño-Pachón, A. M., Sawamoto, K., Stapleton, M., Khan, S., Piechnik, M., Álvarez, J. V., & Tomatsu, S. (2024). Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice. Human gene therapy, 10.1089/hum.2024.096. Advance online publication. https://doi.org/10.1089/hum.2024.096
Disciplines
Medicine and Health Sciences
PubMedID
39450470
Department(s)
Department of Psychiatry, Fellows and Residents
Document Type
Article
COinS