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

• Published in: Human gene therapy Vol. 15; no. 11; p. 1131
• Main Authors: 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
• Format: Journal Article
• Language: English
• Published: United States 01.11.2004
• Subjects: Adolescent; Aminopeptidases; Animals; Brain - metabolism; Cercopithecus aethiops; Child; Child, Preschool; Dependovirus - genetics; Dipeptidyl-Peptidases and Tripeptidyl-Peptidases; DNA, Complementary - metabolism; Endopeptidases - genetics; Gene Transfer Techniques; Genetic Therapy - methods; Genetic Vectors; Glycosylation; Humans; Male; Models, Genetic; Mutation; Neuronal Ceroid-Lipofuscinoses - metabolism; Open Reading Frames; Peptide Hydrolases - genetics; Prospective Studies; Rats; Serine Proteases; Time Factors
• ISSN: 1043-0342
• DOI: 10.1089/hum.2004.15.1131

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...