Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA

Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA

Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of N-acetyl-galactosamine-6-sulfate sulfatase (GALNS), leading to the accumulation of keratan sulfate and chondroitin-6-sulfate and development of severe skeletal dysplasia. Enzyme replacement therapy and hematopoietic stem cell tran...

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Bibliographic Details
Main Authors: Shaukat A. Khan, Jose Victor Álvarez, F.N.U. Nidhi, Eliana Benincore-Florez, Shunji Tomatsu
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
gene therapy
tandem promoters
MPS IVA
GALNS
skeletal dysplasia
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000427
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