In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression

Abstract 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 (Galc twi/twi ) mouse, a KD model with a premature termination codon (PTC) caused by a sing...

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Egile Nagusiak:	Bae-Geun Nam, Jung Hwa Seo, Sung-Ah Hong, Ju-Hee Kim, Minju Kang, Geneva Rose Notario, Yoontaik Hong, Seunghee Cho, Sangsu Bae, Sung-Rae Cho
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	BMC 2026-04-01
Saila:	Genome Medicine
Gaiak:	Krabbe disease Galc adenine base editor adeno-associated virus 9 myelination
Sarrera elektronikoa:	https://doi.org/10.1186/s13073-026-01620-2
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