Paul T. Ranum

The paper develops a computational model to predict therapeutic benefits of reducing somatic instability in Huntington's disease and validates it with preclinical experiments.

The paper creates a temporal single-nucleus transcriptomic atlas of Huntington's disease mouse models to identify stage-specific transcriptional changes in corticostriatal regions.

The paper identifies an optimized AAV capsid, AAV-DB-3, that efficiently transduces deep brain and cortical structures in adult nonhuman primates at low doses.

This paper shows that APOE2 gene therapy reduces amyloid deposition, neurodegeneration, and microglial activation in an Alzheimer's mouse model with ongoing APOE4 expression.

This paper introduces a drug-inducible switch system (Xon) for precise control of gene therapies, enabling gene silencing or replacement with oral small molecule inducers that are safe and bioavailable.