Article
In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression.
Genome medicine - 23 Apr 2026
Nam Bae-Geun, Seo Jung Hwa, Hong Sung-Ah, Kim Ju-Hee, Kang Minju, Notario Geneva Rose, Hong Yoontaik, Cho Seunghee, Bae Sangsu, Cho Sung-Rae
Abstract excerpt
BACKGROUND: Krabbe disease (KD) is caused by mutation of the galactosylceramidase (GALC) gene, leading to deficient sphingolipid metabolism, which is essential for functional myelination. The twitcher (Galctwi/twi) mouse, a KD model with a premature termination codon (PTC) caused by a single-nucleotide G-to-A substitution at the 355th codon of the Galc gene, is a model candidate for treatment with adenine base...
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