Nature Communications (Sep 2021)
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
- Joan Bertolin,
- Víctor Sánchez,
- Albert Ribera,
- Maria Luisa Jaén,
- Miquel Garcia,
- Anna Pujol,
- Xavier Sánchez,
- Sergio Muñoz,
- Sara Marcó,
- Jennifer Pérez,
- Gemma Elias,
- Xavier León,
- Carles Roca,
- Veronica Jimenez,
- Pedro Otaegui,
- Francisca Mulero,
- Marc Navarro,
- Jesús Ruberte,
- Fatima Bosch
Affiliations
- Joan Bertolin
- Center of Animal Biotechnology and Gene Therapy
- Víctor Sánchez
- Center of Animal Biotechnology and Gene Therapy
- Albert Ribera
- Center of Animal Biotechnology and Gene Therapy
- Maria Luisa Jaén
- Center of Animal Biotechnology and Gene Therapy
- Miquel Garcia
- Center of Animal Biotechnology and Gene Therapy
- Anna Pujol
- Center of Animal Biotechnology and Gene Therapy
- Xavier Sánchez
- Center of Animal Biotechnology and Gene Therapy
- Sergio Muñoz
- Center of Animal Biotechnology and Gene Therapy
- Sara Marcó
- Center of Animal Biotechnology and Gene Therapy
- Jennifer Pérez
- Center of Animal Biotechnology and Gene Therapy
- Gemma Elias
- Center of Animal Biotechnology and Gene Therapy
- Xavier León
- Center of Animal Biotechnology and Gene Therapy
- Carles Roca
- Center of Animal Biotechnology and Gene Therapy
- Veronica Jimenez
- Center of Animal Biotechnology and Gene Therapy
- Pedro Otaegui
- Center of Animal Biotechnology and Gene Therapy
- Francisca Mulero
- Molecular Imaging Unit, Spanish National Cancer Research Center (CNIO)
- Marc Navarro
- Center of Animal Biotechnology and Gene Therapy
- Jesús Ruberte
- Center of Animal Biotechnology and Gene Therapy
- Fatima Bosch
- Center of Animal Biotechnology and Gene Therapy
- DOI
- https://doi.org/10.1038/s41467-021-25697-y
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 14
Abstract
Mucopolysaccharidosis type IVA (MPSIVA) is a lysosomal storage disorder causing severe skeletal and non-skeletal alterations in patients. Here, the authors generate a MPSIVA rat model that mimics the disabling human pathology and develop an AAV9-Galns gene therapy to treat the disease.
