Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector

Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by a functional deficiency in human arylsulfatase A (hASA). We recently reported that ependymal cells and the choroid plexus are selectively transduced by intracerebroventricular (ICV) injection of adeno-associated virus seroty...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Sci Rep
Prif Awduron: Hironaka, Kohei, Yamazaki, Yoshiyuki, Hirai, Yukihiko, Yamamoto, Motoko, Miyake, Noriko, Miyake, Koichi, Okada, Takashi, Morita, Akio, Shimada, Takashi
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2015
Pynciau:
Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4539541/
https://ncbi.nlm.nih.gov/pubmed/26283284
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep13104
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