Article

Preclinical validation of AAV9-TECPR2 gene therapy in a novel knock-in model of TECPR2-related disorder

2026-03-04

Abstract excerpt

TECPR2 -related disorder is a rare, autosomal recessive neurodevelopmental and neurodegenerative disease characterized by early-onset motor dysfunction, sensory- and autonomic neuropathy, and progressive neurological decline with early mortality. Currently, there are no effective treatments for individuals affected by this debilitating condition. To advance our understanding of disease mechanisms and explore thera...

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Literature Corpus work: 1aa7bc4b-7f1a-55ac-840b-d6a8bbde58ef
DOI: 10.64898/2026.03.02.708636

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