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Aav-ep+.htpp1

Intervention

1 paper3 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
  • Animals1

Outcomes

3

TPP1

upin N. primates1

1 study

  1. Aav-ep+.htpp1 increases TPP1 in N. primates (>30x lower dose).

    “In NHPs, high cerebrospinal fluid (CSF) TPP1 concentrations were achieved after intracerebroventricular delivery of AAV-Ep+.hTPP1 at a total dose of 1 × 1012 viral genomes, which was more than 30× lower than previously reported doses in NHPs.”

    Animalscerebrospinal fluid1 x 1012 viral genomes

    An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice

    Science translational medicine14 May 2025

More on TPP1

Tremor

upin mice1

1 study

  1. Aav-ep+.htpp1 improves tremor in mice.

    “significantly improving tremor and life span”

    Animalslacking TPP1 activitybrain

    An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice

    Science translational medicine14 May 2025

Lifespan

upin mice1

1 study

  1. Aav-ep+.htpp1 extends lifespan in mice.

    “AAV-Ep+ provided robust and therapeutically relevant TPP1 protein concentrations in these mice, significantly improving tremor and life span.”

    Animalslacking TPP1 activitybrain

    An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice

    Science translational medicine14 May 2025

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