Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

  title={Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.},
  author={S. Worgall and D. Sondhi and N. Hackett and B. Kosofsky and M. Kekatpure and N. Neyzi and J. Dyke and D. Ballon and L. Heier and B. Greenwald and P. Christos and M. Mazumdar and M. Souweidane and M. Kaplitt and R. Crystal},
  journal={Human gene therapy},
  volume={19 5},
  • S. Worgall, D. Sondhi, +12 authors R. Crystal
  • Published 2008
  • Medicine
  • Human gene therapy
  • Late infantile neuronal ceroid lipofuscinosis (LINCL) is an autosomal recessive, neurodegenerative lysosomal storage disease affecting the CNS and is fatal by age 8 to 12 years. A total average dose of 2.5 10(12) particle units of an adeno-associated virus (AAV) serotype 2 vector expressing the human CLN2 cDNA (AAV2 CU h-CLN2) was administered to 12 locations in the CNS of 10 children with LINCL. In addition to safety parameters, a neurological rating scale (primary variable) and three… CONTINUE READING
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