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

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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Autores principales: 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
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/4ba8f5aa92cf4b558796ff21512a7699
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spelling oai:doaj.org-article:4ba8f5aa92cf4b558796ff21512a76992021-12-02T14:55:09ZTreatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy10.1038/s41467-021-25697-y2041-1723https://doaj.org/article/4ba8f5aa92cf4b558796ff21512a76992021-09-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-25697-yhttps://doaj.org/toc/2041-1723Mucopolysaccharidosis 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.Joan BertolinVíctor SánchezAlbert RiberaMaria Luisa JaénMiquel GarciaAnna PujolXavier SánchezSergio MuñozSara MarcóJennifer PérezGemma EliasXavier LeónCarles RocaVeronica JimenezPedro OtaeguiFrancisca MuleroMarc NavarroJesús RuberteFatima BoschNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-14 (2021)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
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
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
description 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.
format article
author 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
author_facet 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
author_sort Joan Bertolin
title Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
title_short Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
title_full Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
title_fullStr Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
title_full_unstemmed Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
title_sort treatment of skeletal and non-skeletal alterations of mucopolysaccharidosis type iva by aav-mediated gene therapy
publisher Nature Portfolio
publishDate 2021
url https://doaj.org/article/4ba8f5aa92cf4b558796ff21512a7699
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