Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

@article{Watson2006IntrathecalAO,
  title={Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice},
  author={Geoffrey Norman Watson and Jacob Bastacky and Pavel V. Belichenko and Madhavee M. Buddhikot and Steven J. Jungles and Michel C. Vellard and William C Mobley and E. Kakkis},
  journal={Gene Therapy},
  year={2006},
  volume={13},
  pages={917-925}
}
Mucopolysaccharidosis type I (MPS I) is caused by an inherited deficiency of α-L-iduronidase (IDUA). The result is a progressive, lysosomal storage disease with central nervous system (CNS) as well as systemic involvement. To target gene therapy to the CNS, recombinant adeno-associated virus (AAV) vectors carrying IDUA sequence were administered to MPS I mice via injection into cerebrospinal fluid. In contrast to intravenous administration, this intrathecal administration was effective in… CONTINUE READING

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Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector.

Molecular therapy : the journal of the American Society of Gene Therapy • 2005

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