Author:
Hocquemiller Michaël,Giersch Laura,Mei Xin,Gross Amanda L.,Randle Ashley N.,Gray-Edwards Heather L.,Hudson Judith A.,Todeasa Sophia,Stoica Lorelei,Martin Douglas R.,Sena-Esteves Miguel,Aiach Karen,Laufer Ralph
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference38 articles.
1. GM1 gangliosidosis: review of clinical, molecular, and therapeutic aspects;Brunetti-Pierri;Mol. Genet. Metab.,2008
2. GLB1-Related Disorders, in GeneReviews((R));Regier,2013
3. The GM1 and GM2 gangliosidoses: natural history and progress toward therapy;Regier;Pediatr. Endocrinol. Rev.,2016
4. AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival;Baek;PLoS One,2010
5. Lipidomic evaluation of feline neurologic disease after AAV gene therapy;Gray-Edwards;Mol. Ther. Methods Clin. Dev.,2017
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