TPP1

1 paper1 finding

Animals1

Interventions

1

Aav-ep+.htpp1

upin N. primates1

1 study

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

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Science Translational Medicine14 May

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

Experiments in miceInterventions