Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10

Sanfilippo syndrome type B (mucopolysaccharidosis IIIB, MPS IIIB) is a lysosomal storage disease resulting from deficiency of N-acetyl-glucosaminidase (NAGLU) activity. To determine the possible therapeutic utility of recombinant adeno-associated virus (rAAV) in early gene therapy-based intervention...

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Detalles Bibliográficos
Autores principales: Gilkes, J A, Bloom, M D, Heldermon, C D
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4777635/
https://www.ncbi.nlm.nih.gov/pubmed/26674264
http://dx.doi.org/10.1038/gt.2015.111

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4777635/
https://www.ncbi.nlm.nih.gov/pubmed/26674264
http://dx.doi.org/10.1038/gt.2015.111

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