Beverly L. Davidson

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 develops humanized mouse models for SYNGAP1 and STXBP1 disorders, showing similar electrophysiological and behavioral abnormalities in mice and patients, and demonstrates the efficacy of antisense oligonucleotides in modulating SYNGAP1 expression.

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

This paper develops novel AAV capsids with improved transduction efficiency for airway epithelial cells, reducing required doses and enhancing therapeutic potential for respiratory diseases.

This paper demonstrates that CRISPR-mediated transcriptional activation can upregulate SYNGAP1 in a mouse model of SRID and human neurons, rescuing deficits without targeting mutations.

The paper uses MBNL-dependent alternative splicing cassette exons to control gene therapy in myotonic dystrophy by tuning therapeutic protein translation.

The paper identifies chaperones in the human Hsp70 network that buffer TDP-43 toxicity by reducing aggregates and enhancing solubility.

The paper uses a CRISPR screen to identify genes affecting rAAV production in HEK 293 cells, finding that heparan sulfate biosynthesis and vesicular trafficking genes influence yield.

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.

The paper demonstrates that CRISPR-Cas9 nickase can contract CAG/CTG repeats in patient-derived cells and improve molecular and behavioral phenotypes in Huntington's disease.

The paper investigates the roles of canonical and alternative RPA in CAG repeat diseases, finding that Alt-RPA antagonizes RPA's suppression of CAG expansions, contributing to disease pathogenesis.

This study shows that restoring Cln3 in neurons normalizes brain network dysfunction in CLN3 disease despite persistent storage accumulation, suggesting neuronal loss-of-function, not storage, drives neurologic symptoms.

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.

This study shows that AAV-mediated GRN overexpression in mice causes hippocampal degeneration and T cell infiltration, raising safety concerns for GRN-based CNS gene therapies.