AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease
Author:
Funder
Mila’s Miracle Foundation and Batten Hope Foundation
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://www.jci.org/articles/view/146286/files/pdf
Reference64 articles.
1. The Novel Neuronal Ceroid Lipofuscinosis Gene MFSD8 Encodes a Putative Lysosomal Transporter
2. Genetics of the neuronal ceroid lipofuscinoses (Batten disease);Mole;Biochim Biophys Acta,2015
3. Major Facilitator Superfamily
4. Expression and lysosomal targeting of CLN7, a major facilitator superfamily transporter associated with variant late-infantile neuronal ceroid lipofuscinosis
5. Gene disruption of Mfsd8 in mice provides the first animal model for CLN7 disease
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