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

@article{Worgall2008TreatmentOL,
  title={Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.},
  author={Stefan Worgall and Dolan Sondhi and Neil R. Hackett and B. Kosofsky and Minal V Kekatpure and Nurunisa Neyzi and Jonathan P. Dyke and Douglas J. Ballon and Linda A. Heier and Bruce M. Greenwald and Paul J. Christos and Madhu Mazumdar and M M Souweidane and Michael G Kaplitt and Ronald G. Crystal},
  journal={Human gene therapy},
  year={2008},
  volume={19 5},
  pages={
          463-74
        }
}
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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