The Effect of Neonatal Gene Therapy on Skeletal Manifestations in Mucopolysaccharidosis VII Dogs after a Decade

Mucopolysaccharidosis (MPS) VII is a lysosomal storage disease due to deficient activity of β-glucuronidase (GUSB), and results in glycosaminoglycan accumulation. Skeletal manifestations include bone dysplasia, degenerative joint disease, and growth retardation. One gene therapy approach for MPS VII...

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Главные авторы: Xing, Elizabeth M., Knox, Van W., O'Donnell, Patricia A., Sikura, Tracey, Liu, Yuli, Wu, Susan, Casal, Margret L., Haskins, Mark E., Ponder, Katherine P.
Формат: Artigo
Язык:Inglês
Опубликовано: 2013
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Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3690974/
https://ncbi.nlm.nih.gov/pubmed/23628461
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2013.03.013
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