Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy

Sulfamidase (SGSH) deficiency causes mucopolysaccharidosis type IIIA (MPS IIIA), a lysosomal storage disease (LSD) that affects the CNS. In earlier work in LSD mice and dog models, we exploited the utility of adeno-associated viruses (AAVs) to transduce brain ventricular lining cells (ependyma) for...

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Udgivet i:Mol Ther
Main Authors: Chen, Yonghong, Zheng, Shujuan, Tecedor, Luis, Davidson, Beverly L.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6079371/
https://ncbi.nlm.nih.gov/pubmed/29503202
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.01.010
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