scholarly journals AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease

2022 ◽  
Author(s):  
Xin Chen ◽  
Thomas Dong ◽  
Yuhui Hu ◽  
Frances C. Shaffo ◽  
Nandkishore R. Belur ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Neuronal Ceroid Lipofuscinosis ◽  
Preclinical Models ◽  
Ceroid Lipofuscinosis ◽  
Mfsd8 Gene

A novel mutation in the MFSD8 gene in late infantile neuronal ceroid lipofuscinosis

Neurogenetics ◽  
2008 ◽  
Vol 10 (1) ◽  
pp. 73-77 ◽  
Author(s):  
E. Stogmann ◽  
S. El Tawil ◽  
J. Wagenstaller ◽  
A. Gaber ◽  
S. Edris ◽  
...  
Keyword(s):  
Neuronal Ceroid Lipofuscinosis ◽  
Novel Mutation ◽  
Infantile Neuronal Ceroid Lipofuscinosis ◽  
Ceroid Lipofuscinosis ◽  
Mfsd8 Gene
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