Luis Tecedor

Computational modeling and preclinical validation support targeting somatic instability for Huntington's disease treatment

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

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.

Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation

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.

Regulated control of gene therapies with a drug induced switch

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.