Response to Letter to the Editor: Secondary ganglioside GM2 accumulation in mucopolysaccharidoses
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference5 articles.
1. Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells;Ross;Hum. Gene Ther.,2000
2. Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse;Sferra;Mol. Ther.,2004
3. Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer;McIntrye;Gene Med,2010
4. A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome);Bhaumik;Glycobiology,1999
5. Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model;Martins;Brain,2015
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