Antisense oligonucleotide therapy for KCNT1 encephalopathy.
Burbano LE, Li M, Jancovski N, Jafar-Nejad P, Richards K, Sedo A, Soriano A, Rollo B, Jia L, Gazina EV, Piltz S, Adikusuma F, Thomas PQ, Kopsidas H, Rigo F, Reid CA, Maljevic S, Petrou S.
Burbano LE, et al.
JCI Insight. 2022 Dec 8;7(23):e146090. doi: 10.1172/jci.insight.146090.
JCI Insight. 2022.
PMID: 36173683
Free PMC article.