Article
Mono- and Biallelic Inactivation of Huntingtin Gene in Patient-Specific Induced Pluripotent Stem Cells Reveal HTT Roles in Striatal Development and Neuronal Functions.
Journal of Huntington's disease - 1 Jan 2024
Louessard Morgane, Cailleret Michel, Jarrige Margot, Bigarreau Julie, Lenoir Sophie, Dufour Noëlle, Rey Maria, Saudou Frédéric, Deglon Nicole, Perrier Anselme L
Abstract excerpt
Background: Mutations in the Huntingtin (HTT) gene cause Huntington's disease (HD), a neurodegenerative disorder. As a scaffold protein, HTT is involved in numerous cellular functions, but its normal and pathogenic functions during human forebrain development are poorly understood. Objective: To investigate the developmental component of HD, with a specific emphasis on understanding the functions of wild-type and...
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