Disease correction by Aav-mediated gene therapy in a new mouse model of mucopolysaccharidosis type Iiid

Carles Roca; Sandra Motas; Sara Marcó; Albert Ribera; Víctor Sánchez; Xavier Sánchez; Joan Bertolin; Xavier León; Jennifer Pérez; Miguel Garcia; Pilar Villacampa; Jesús Ruberte; Anna Pujol; Virginia Haurigot; Fatima Bosch

doi:10.1093/hmg/ddx058

DOI: 10.1093/hmg/ddx058

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PMID: 28334745

Issn Print: 0964-6906

Publication Date: 2017/04/15

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Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID

Carles Roca; Sandra Motas; Sara Marcó; Albert Ribera; Víctor Sánchez; Xavier Sánchez; Joan Bertolin; Xavier León; Jennifer Pérez; Miguel Garcia; Pilar Villacampa; Jesús Ruberte; Anna Pujol; Virginia Haurigot; Fatima Bosch

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Author Information: Center of Animal Biotechnology and Gene Therapy


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