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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. Among authors: rigo f. JCI Insight. 2022 Dec 8;7(23):e146090. doi: 10.1172/jci.insight.146090. JCI Insight. 2022. PMID: 36173683 Free PMC article.
Therapeutic inhibition of mTORC2 rescues the behavioral and neurophysiological abnormalities associated with Pten-deficiency.
Chen CJ, Sgritta M, Mays J, Zhou H, Lucero R, Park J, Wang IC, Park JH, Kaipparettu BA, Stoica L, Jafar-Nejad P, Rigo F, Chin J, Noebels JL, Costa-Mattioli M. Chen CJ, et al. Among authors: rigo f. Nat Med. 2019 Nov;25(11):1684-1690. doi: 10.1038/s41591-019-0608-y. Epub 2019 Oct 21. Nat Med. 2019. PMID: 31636454 Free PMC article.
A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis.
Rodriguez CM, Wright SE, Kearse MG, Haenfler JM, Flores BN, Liu Y, Ifrim MF, Glineburg MR, Krans A, Jafar-Nejad P, Sutton MA, Bassell GJ, Parent JM, Rigo F, Barmada SJ, Todd PK. Rodriguez CM, et al. Among authors: rigo f. Nat Neurosci. 2020 Mar;23(3):386-397. doi: 10.1038/s41593-020-0590-1. Epub 2020 Feb 17. Nat Neurosci. 2020. PMID: 32066985 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. Among authors: rigo f. Nucleic Acids Res. 2021 Jan 25;49(2):657-673. doi: 10.1093/nar/gkaa1235. Nucleic Acids Res. 2021. PMID: 33367834 Free PMC article.
432 results