Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse

Mucopolysaccharidosis type IIIB (MPS IIIB) is an autosomal recessive lysosomal disease caused by defective production of the enzyme α-N-acetylglucosaminidase. It is characterized by severe and complex central nervous system degeneration. Effective therapies will likely target early onset disease and...

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Detalhes bibliográficos
Publicado no:Gene Ther
Main Authors: Gilkes, Janine A., Judkins, Benjamin L., Herrera, Brontie N., Mandel, Ronald J., Boye, Sanford L., Boye, Shannon E., Srivastava, Arun, Heldermon, Coy D.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8149485/
https://ncbi.nlm.nih.gov/pubmed/33244179
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-020-00206-w
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