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Aav2/5-ppt1

Intervention

1 paper2 findings

C, W, M, H: cells, worms or flies, mice, humans. Dark: independent labs found the same change there. Light: one lab or one study. Outline: nothing yet.

  • 1 lab in 1 country
  • Funders: National Institute of Neurological Disorders and Stroke and 1 more
  • Animals1

Outcomes

2

Motor function

upin mice1

1 study

  1. Aav2/5-ppt1 improves motor function in mice.

    “AAV2/5-mediated gene therapy alone resulted in significant histological correction, improved motor function, and increased lifespan.”

    AnimalsPpt1(-/-) mice

    Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

    Annals of neurology24 Feb 2012

Lifespan

upin mice1

1 study

  1. Aav2/5-ppt1 extends lifespan in mice.

    “AAV2/5-mediated gene therapy alone resulted in significant histological correction, improved motor function, and increased lifespan.”

    AnimalsPpt1(-/-) mice

    Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

    Annals of neurology24 Feb 2012

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