Neonatal Gene Therapy With a Gamma Retroviral Vector in Mucopolysaccharidosis VI Cats

Mucopolysaccharidosis (MPS) VI is due to a deficiency in the activity of N-acetylgalactosamine 4-sulfatase (4S), also known as arylsulfatase B. Previously, retroviral vector (RV)-mediated neonatal gene therapy reduced the clinical manifestations of MPS I and MPS VII in mice and dogs. However, sulfat...

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Bibliographic Details
Main Authors: Ponder, Katherine P, O'Malley, Thomas M, Wang, Ping, O'Donnell, Patricia A, Traas, Anne M, Knox, Van W, Aguirre, Gustavo A, Ellinwood, N Matthew, Metcalf, Jason A, Wang, Bin, Parkinson-Lawrence, Emma J, Sleeper, Meg M, Brooks, Doug A, Hopwood, John J, Haskins, Mark E
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2012
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3345988/
https://ncbi.nlm.nih.gov/pubmed/22395531
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.9
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