209. Correction of Hunter Syndrome in the MPSII Mouse Model by AAV2/8-Mediated Gene Delivery
Year: 2006
Type: article
Abstract: Mucopolysaccharidosis type II (MPSII; Hunter syndrome) is a lysosomal storage disorder caused by a deficiency in the enzyme iduronate 2-sulfatase. At present, the therapeutic approaches for MPSII are enzyme replacement therapy and bone marrow transplantation, although these therapies have some limitations. The availability of new AAV serotypes that display tissue-specific tropism and promote sustained expression of transgenes offers the possibility of AAV-mediated gene therapy for the systemic treatment of lysosomal diseases, including MPSII.
Source: Molecular Therapy
Authors: Monica Cardone, Vinicia Assunta Polito, Stefano Pepe, Linda Mann, Alessandra D'Azzo, Alberto Auricchio, Andrea Ballabio, Maria Pia Cosma
Language: English
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W4249398170