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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.
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.
Jafar-Nejad P, Powers B, Soriano A, Zhao H, Norris DA, Matson J, DeBrosse-Serra B, Watson J, Narayanan P, Chun SJ, Mazur C, Kordasiewicz H, Swayze EE, Rigo F. Jafar-Nejad P, et al. Nucleic Acids Res. 2021 Jan 25;49(2):657-673. doi: 10.1093/nar/gkaa1235. Nucleic Acids Res. 2021. PMID: 33367834 Free PMC article.
52 results