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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Detalles Bibliográficos
Autores principales: Chen, Yonghong, Zheng, Shujuan, Tecedor, Luis, Davidson, Beverly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6079371/
https://www.ncbi.nlm.nih.gov/pubmed/29503202
http://dx.doi.org/10.1016/j.ymthe.2018.01.010

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6079371/
https://www.ncbi.nlm.nih.gov/pubmed/29503202
http://dx.doi.org/10.1016/j.ymthe.2018.01.010

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