Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency, resulting in keratan sulfate (KS) and chondroitin-6-sulfate accumulation. Patients develop severe skeletal dysplasia, early cartilage...

Descripción completa

Detalles Bibliográficos
Autores principales: Bertolin, Joan, Sánchez, Víctor, Ribera, Albert, Jaén, Maria Luisa, Garcia, Miquel, Pujol, Anna, Sánchez, Xavier, Muñoz, Sergio, Marcó, Sara, Pérez, Jennifer, Elias, Gemma, León, Xavier, Roca, Carles, Jimenez, Veronica, Otaegui, Pedro, Mulero, Francisca, Navarro, Marc, Ruberte, Jesús, Bosch, Fatima
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8429698/
https://www.ncbi.nlm.nih.gov/pubmed/34504088
http://dx.doi.org/10.1038/s41467-021-25697-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8429698/
https://www.ncbi.nlm.nih.gov/pubmed/34504088
http://dx.doi.org/10.1038/s41467-021-25697-y

Ejemplares similares