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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: soriano a. Nucleic Acids Res. 2021 Jan 25;49(2):657-673. doi: 10.1093/nar/gkaa1235. Nucleic Acids Res. 2021. PMID: 33367834 Free PMC article.
Comprehensive Structure-Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes.
Prakash TP, Yu J, Migawa MT, Kinberger GA, Wan WB, Østergaard ME, Carty RL, Vasquez G, Low A, Chappell A, Schmidt K, Aghajan M, Crosby J, Murray HM, Booten SL, Hsiao J, Soriano A, Machemer T, Cauntay P, Burel SA, Murray SF, Gaus H, Graham MJ, Swayze EE, Seth PP. Prakash TP, et al. Among authors: soriano a. J Med Chem. 2016 Mar 24;59(6):2718-33. doi: 10.1021/acs.jmedchem.5b01948. Epub 2016 Mar 9. J Med Chem. 2016. PMID: 26914862
Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice.
Becker LA, Huang B, Bieri G, Ma R, Knowles DA, Jafar-Nejad P, Messing J, Kim HJ, Soriano A, Auburger G, Pulst SM, Taylor JP, Rigo F, Gitler AD. Becker LA, et al. Among authors: soriano a. Nature. 2017 Apr 20;544(7650):367-371. doi: 10.1038/nature22038. Epub 2017 Apr 12. Nature. 2017. PMID: 28405022 Free PMC article.
Antisense oligonucleotide therapy reduces seizures and extends life span in an SCN2A gain-of-function epilepsy model.
Li M, Jancovski N, Jafar-Nejad P, Burbano LE, Rollo B, Richards K, Drew L, Sedo A, Heighway J, Pachernegg S, Soriano A, Jia L, Blackburn T, Roberts B, Nemiroff A, Dalby K, Maljevic S, Reid CA, Rigo F, Petrou S. Li M, et al. Among authors: soriano a. J Clin Invest. 2021 Dec 1;131(23):e152079. doi: 10.1172/JCI152079. J Clin Invest. 2021. PMID: 34850743 Free PMC article.
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: soriano a. JCI Insight. 2022 Dec 8;7(23):e146090. doi: 10.1172/jci.insight.146090. JCI Insight. 2022. PMID: 36173683 Free PMC article.
Reduction of nemo-like kinase increases lysosome biogenesis and ameliorates TDP-43-related neurodegeneration.
Tejwani L, Jung Y, Kokubu H, Sowmithra S, Ni L, Lee C, Sanders B, Lee PJ, Xiang Y, Luttik K, Soriano A, Yoon J, Park J, Ro HH, Ju H, Liao C, Tieze SM, Rigo F, Jafar-Nejad P, Lim J. Tejwani L, et al. Among authors: soriano a. J Clin Invest. 2023 Aug 15;133(16):e138207. doi: 10.1172/JCI138207. J Clin Invest. 2023. PMID: 37384409 Free PMC article.
929 results