Ellie M. Carrell

Co-opting MBNL-dependent alternative splicing cassette exons to control gene therapy in myotonic dystrophy

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

Optimized AAV capsids for diseases of the basal ganglia show robust potency and distribution in adult nonhuman primates

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.

APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease

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