Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector

No treatment is available to address the unmet needs of mucopolysaccharidosis (MPS) IIIA patients. Targeting the root cause, we developed a new self-complementary adeno-associated virus 9 (scAAV9) vector to deliver the human N-sulfoglucosamine sulfohydrolase (hSGSH) gene driven by a miniature cytome...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Bobo, Tierra A., Samowitz, Preston N., Robinson, Michael I., Fu, Haiyan
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7704409/
https://ncbi.nlm.nih.gov/pubmed/33313335
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.10.014
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