Article
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.
Human gene therapy - 1 Nov 2004
Crystal Ronald G, Sondhi Dolan, Hackett Neil R, Kaminsky Stephen M, Worgall Stefan, Stieg Philip, Souweidane Mark, Hosain Syed, Heier Linda, Ballon Douglas, Dinner Miles, Wisniewski Krystyna, Kaplitt Michael, Greenwald Bruce M, Howell Joy D, Strybing Kristin, Dyke Jonathan, Voss Henning
Abstract excerpt
Late infantile neuronal ceroid lipofuscinosis (LINCL) is a fatal childhood neurodegenerative lysosomal storage disease with no known therapy. There are estimated to be 200 to 300 children in the United States at any one time with the disease. LINCL is a genetic disease resulting from a deficiency of tripeptidyl peptidase I (TPP-I), a proteolytic enzyme encoded by CLN2, the gene that is mutated in individuals with...
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