Intrastriatal delivery of a zinc finger protein targeting the mutant HTT gene by AAV9 obviates lipid phenotypes in brain and plasma of zQ175DN HD mice
Andrew Iwanowicz, Adel Boudi, Connor Seeley, Ellen Sapp, Rachael Miller, Sophia Liu, Kathryn O. Chase, Kai Shing, Ana Rita Batista, Miguel Sena‐Esteves, Neil Aronin M. D, Marian DiFiglia, Kimberly B. Kegel-Gleason
The paper shows that reducing mutant HTT with a zinc finger protein in HD mice prevents lipid changes in brain and plasma.
bioRxiv · Jan 6, 2025 · PreprintNeuroscience