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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Detalles Bibliográficos
Autores principales: Gilkes, Janine A., Judkins, Benjamin L., Herrera, Brontie N., Mandel, Ronald J., Boye, Sanford L., Boye, Shannon E., Srivastava, Arun, Heldermon, Coy D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8149485/
https://www.ncbi.nlm.nih.gov/pubmed/33244179
http://dx.doi.org/10.1038/s41434-020-00206-w

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8149485/
https://www.ncbi.nlm.nih.gov/pubmed/33244179
http://dx.doi.org/10.1038/s41434-020-00206-w

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