Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
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.
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Auteurs principaux: | , , , , , , , , , , , , , , , , , , |
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Format: | article |
Langue: | EN |
Publié: |
Nature Portfolio
2021
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Sujets: | |
Accès en ligne: | https://doaj.org/article/4ba8f5aa92cf4b558796ff21512a7699 |
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