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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Udgivet i: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.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2020
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Article
Online adgang: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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