Preferred transduction with AAV8 and AAV9 via thalamic administration in the MPS IIIB model: A comparison of four rAAV serotypes

Sanfilippo syndrome type B (MPS IIIB) is a lysosomal storage disease caused by a deficiency of N-acetyl-glucosaminidase (NAGLU) activity. Since early therapeutic intervention is likely to yield the most efficacious results, we sought to determine the possible therapeutic utility of rAAV in early gen...

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Detalles Bibliográficos
Autores principales: Gilkes, J.A., Bloom, M.D., Heldermon, C.D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4789330/
https://www.ncbi.nlm.nih.gov/pubmed/27014573
http://dx.doi.org/10.1016/j.ymgmr.2015.11.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4789330/
https://www.ncbi.nlm.nih.gov/pubmed/27014573
http://dx.doi.org/10.1016/j.ymgmr.2015.11.006

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