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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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| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Nature Publishing Group
2012
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| Fag: | |
| Online adgang: | 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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