Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis
Author:
Publisher
Elsevier BV
Subject
Developmental Neuroscience,Neurology
Reference58 articles.
1. New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy;Beck;Hum.Genet.,2007
2. Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration;Bostick;Gene Ther.,2007
3. Enzyme replacement for lysosomal diseases;Brady;Annu. Rev. Med.,2006
4. Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for wide-spread gene delivery to the neonatal mouse brain;Broekman;Neuroscience,2006
5. Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery;Broekman;Mol. Ther.,2007
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