Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice

Mucopolysaccharidosis type IIIB (MPS IIIB) or Sanfilippo Syndrome type B is a lysosomal storage disease resulting from the deficiency of N-acetyl glucosaminidase (NAGLU) activity. We previously showed that intracranial adeno-associated virus (AAV) -based gene therapy results in partial improvements...

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Main Authors: Heldermon, Coy D., Qin, Elizabeth Y., Ohlemiller, Kevin K., Herzog, Erik D., Brown, Jillian R., Vogler, Carol, Hou, Wei, Orrock, John L., Crawford, Brett E., Sands, Mark S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2013
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3701029/
https://ncbi.nlm.nih.gov/pubmed/23535899
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2013.14
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