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.

@article{Crystal2004ClinicalPA,
  title={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.},
  author={Ronald G. Crystal and D Sondhi and Neil R. Hackett and S M Kaminsky and Stefan Worgall and Philip Edwin Stieg and M M Souweidane and Syed A Hosain and Linda A. Heier and Douglas J. Ballon and Miles Dinner and Krystyna E. Wisniewski and Michael G. Kaplitt and Bruce M Greenwald and Joy D Howell and Kristin Strybing and Jonathan P. Dyke and Henning Voss},
  journal={Human gene therapy},
  year={2004},
  volume={15 11},
  pages={1131-54}
}
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 LINCL. The subjects are chronically ill, with a progressive CNS disorder that… CONTINUE READING