Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB

Sanfilippo syndrome type B (MPS IIIB) is a lysosomal storage disease resulting from a deficiency of N-acetyl-glucosaminidase (NAGLU) activity. In an attempt to correct the disease in the murine model of MPS IIIB, neonatal mice were treated with intracranial AAV2/5-NAGLU (AAV), syngeneic bone marrow...

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Detalhes bibliográficos
Main Authors: Heldermon, Coy D, Ohlemiller, Kevin K, Herzog, Erik D, Vogler, Carole, Qin, Elizabeth, Wozniak, David F, Tan, Yun, Orrock, John L, Sands, Mark S
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2010
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Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2890104/
https://ncbi.nlm.nih.gov/pubmed/20179679
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.17
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