MechanismsAnimals
Heterozygosity of lysosomal enzyme genes and Alzheimer's disease
AAIC 2025 Abstract
Abstract
Background: Although lysosome dysfunction has been implicated in Alzheimer's disease (AD), it is unclear what level or type of dysfunction is pathogenic. We hypothesize that haploinsufficiency of lysosomal enzymes is associated with AD and that CNS‐directed, AAV‐mediated gene transfer can be an effective treatment.
The paper
Mark S. Sands, Bruno A. Benítez, Clare E. Wallace, John R. Cirrito
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Maulikkumar Patel, Niko-Petteri Nykänen, Carla M. Yuede, Samantha Eaton, Cyril P. Pottier, Arda Cetin, Matthew Johnson, Mia T. Bevan, Woodrow D. Gardiner, Hannah Margaret Edwards, Brookelyn M. Doherty, Ryan Harrigan, Dominic Kurian, Thomas M. Wishart, Colin Smith, Neal C. Goodwin,Washington University School of Medicine · Washington University in St. Louis
AAIC 2025 Abstract · 23 Dec 2025 · CC BY
Presented at AAIC 2025
