NeurosciencebioRxivPreprint
Postnatal gene restoration in succinic semialdehyde dehydrogenase deficiency (SSADHD) reveals phenotype reversibility
H. Lee, Gabrielle E. McGinty, Amanda Liebhardt…·Alexander Rotenberg
This study shows that restoring the ALDH5A1 gene in a mouse model of SSADHD reverses hyperactivity and reduces GHB levels, improving survival.
DOI 10.64898/2026.03.24.713250