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AAV9-EF1A-NPC1

Intervention

1 paper1 finding

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 Human Genome Research Institute and 1 more
  • Animals1

Outcomes

1

Lifespan

upin mice1

1 study

  1. AAV9-EF1A-NPC1 extends lifespan in mice (from 69 days to 166 days).

    “Treatment with AAV9-EF1a-NPC1, as compared to AAV9-CamKII-NPC1, resulted in significantly increased survival (mean survival increased from 69 days to 166 and 97 days, respectively)”

    Animalsin Npc1-/- miceAAV9-EF1a-NPC11.2 × 1012GC

    Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

    Human molecular genetics1 Jan 2017

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