Rigo F - Search Results

1

Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice.

Lentz JJ, Pan B, Ponnath A, Tran CM, Nist-Lund C, Galvin A, Goldberg H, Robillard KN, Jodelka FM, Farris HE, Huang J, Chen T, Zhu H, Zhou W, Rigo F, Hastings ML, Géléoc GSG. Lentz JJ, et al. Among authors: rigo f. Mol Ther. 2020 Dec 2;28(12):2662-2676. doi: 10.1016/j.ymthe.2020.08.002. Epub 2020 Aug 5. Mol Ther. 2020. PMID: 32818431 Free PMC article.

2

Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness.

Lentz JJ, Jodelka FM, Hinrich AJ, McCaffrey KE, Farris HE, Spalitta MJ, Bazan NG, Duelli DM, Rigo F, Hastings ML. Lentz JJ, et al. Among authors: rigo f. Nat Med. 2013 Mar;19(3):345-50. doi: 10.1038/nm.3106. Epub 2013 Feb 4. Nat Med. 2013. PMID: 23380860 Free PMC article.

3

Therapeutic correction of ApoER2 splicing in Alzheimer's disease mice using antisense oligonucleotides.

Hinrich AJ, Jodelka FM, Chang JL, Brutman D, Bruno AM, Briggs CA, James BD, Stutzmann GE, Bennett DA, Miller SA, Rigo F, Marr RA, Hastings ML. Hinrich AJ, et al. Among authors: rigo f. EMBO Mol Med. 2016 Apr 1;8(4):328-45. doi: 10.15252/emmm.201505846. EMBO Mol Med. 2016. PMID: 26902204 Free PMC article.

4

Antisense oligonucleotides delivered to the amniotic cavity in utero modulate gene expression in the postnatal mouse.

Depreux FF, Wang L, Jiang H, Jodelka FM, Rosencrans RF, Rigo F, Lentz JJ, Brigande JV, Hastings ML. Depreux FF, et al. Among authors: rigo f. Nucleic Acids Res. 2016 Nov 16;44(20):9519-9529. doi: 10.1093/nar/gkw867. Epub 2016 Sep 28. Nucleic Acids Res. 2016. PMID: 27683224 Free PMC article.

5

Rescue of peripheral vestibular function in Usher syndrome mice using a splice-switching antisense oligonucleotide.

Vijayakumar S, Depreux FF, Jodelka FM, Lentz JJ, Rigo F, Jones TA, Hastings ML. Vijayakumar S, et al. Among authors: rigo f. Hum Mol Genet. 2017 Sep 15;26(18):3482-3494. doi: 10.1093/hmg/ddx234. Hum Mol Genet. 2017. PMID: 28633508 Free PMC article.

6

Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment.

Ponnath A, Depreux FF, Jodelka FM, Rigo F, Farris HE, Hastings ML, Lentz JJ. Ponnath A, et al. Among authors: rigo f. J Assoc Res Otolaryngol. 2018 Feb;19(1):1-16. doi: 10.1007/s10162-017-0640-x. Epub 2017 Oct 12. J Assoc Res Otolaryngol. 2018. PMID: 29027038 Free PMC article.

7

Targeting Amyloid-β Precursor Protein, APP, Splicing with Antisense Oligonucleotides Reduces Toxic Amyloid-β Production.

Chang JL, Hinrich AJ, Roman B, Norrbom M, Rigo F, Marr RA, Norstrom EM, Hastings ML. Chang JL, et al. Among authors: rigo f. Mol Ther. 2018 Jun 6;26(6):1539-1551. doi: 10.1016/j.ymthe.2018.02.029. Epub 2018 Mar 6. Mol Ther. 2018. PMID: 29628304 Free PMC article.

8

Delivery of Antisense Oligonucleotides to the Cornea.

Chau VQ, Hu J, Gong X, Hulleman JD, Ufret-Vincenty RL, Rigo F, Prakash TP, Corey DR, Mootha VV. Chau VQ, et al. Among authors: rigo f. Nucleic Acid Ther. 2020 Aug;30(4):207-214. doi: 10.1089/nat.2019.0838. Epub 2020 Mar 23. Nucleic Acid Ther. 2020. PMID: 32202944 Free PMC article.

9

Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction.

Wang L, Kempton JB, Jiang H, Jodelka FM, Brigande AM, Dumont RA, Rigo F, Lentz JJ, Hastings ML, Brigande JV. Wang L, et al. Among authors: rigo f. Nucleic Acids Res. 2020 May 21;48(9):5065-5080. doi: 10.1093/nar/gkaa194. Nucleic Acids Res. 2020. PMID: 32249312 Free PMC article.

10

Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptides.

Nikan M, Tanowitz M, Dwyer CA, Jackson M, Gaus HJ, Swayze EE, Rigo F, Seth PP, Prakash TP. Nikan M, et al. Among authors: rigo f. J Med Chem. 2020 Aug 13;63(15):8471-8484. doi: 10.1021/acs.jmedchem.0c00840. Epub 2020 Aug 2. J Med Chem. 2020. PMID: 32677436

11

Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease.

Centa JL, Jodelka FM, Hinrich AJ, Johnson TB, Ochaba J, Jackson M, Duelli DM, Weimer JM, Rigo F, Hastings ML. Centa JL, et al. Among authors: rigo f. Nat Med. 2020 Sep;26(9):1444-1451. doi: 10.1038/s41591-020-0986-1. Epub 2020 Jul 27. Nat Med. 2020. PMID: 32719489 Free PMC article.

12

Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1.

Ait Benichou S, Jauvin D, De Serres-Bérard T, Pierre M, Ling KK, Bennett CF, Rigo F, Gourdon G, Chahine M, Puymirat J. Ait Benichou S, et al. Among authors: rigo f. Gene Ther. 2022 Dec;29(12):698-709. doi: 10.1038/s41434-022-00316-7. Epub 2022 Jan 25. Gene Ther. 2022. PMID: 35075265 Free PMC article.

13

Corticosteroid sensitization drives opioid addiction.

Carmack SA, Vendruscolo JCM, Adrienne McGinn M, Miranda-Barrientos J, Repunte-Canonigo V, Bosse GD, Mercatelli D, Giorgi FM, Fu Y, Hinrich AJ, Jodelka FM, Ling K, Messing RO, Peterson RT, Rigo F, Edwards S, Sanna PP, Morales M, Hastings ML, Koob GF, Vendruscolo LF. Carmack SA, et al. Among authors: rigo f. Mol Psychiatry. 2022 May;27(5):2492-2501. doi: 10.1038/s41380-022-01501-1. Epub 2022 Mar 16. Mol Psychiatry. 2022. PMID: 35296810 Free PMC article.

14

Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1.

Ait Benichou S, Jauvin D, De Serres-Bérard T, Bennett F, Rigo F, Gourdon G, Boutjdir M, Chahine M, Puymirat J. Ait Benichou S, et al. Among authors: rigo f. Hum Gene Ther. 2022 Aug;33(15-16):810-820. doi: 10.1089/hum.2022.069. Hum Gene Ther. 2022. PMID: 35794764

15

Antisense oligonucleotide-based therapies for diseases caused by pre-mRNA processing defects.

Rigo F, Seth PP, Bennett CF. Rigo F, et al. Adv Exp Med Biol. 2014;825:303-52. doi: 10.1007/978-1-4939-1221-6_9. Adv Exp Med Biol. 2014. PMID: 25201110 Review.

16

Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy.

Bogdanik LP, Osborne MA, Davis C, Martin WP, Austin A, Rigo F, Bennett CF, Lutz CM. Bogdanik LP, et al. Among authors: rigo f. Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5863-72. doi: 10.1073/pnas.1509758112. Epub 2015 Oct 12. Proc Natl Acad Sci U S A. 2015. PMID: 26460027 Free PMC article.

17

Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides.

Sztainberg Y, Chen HM, Swann JW, Hao S, Tang B, Wu Z, Tang J, Wan YW, Liu Z, Rigo F, Zoghbi HY. Sztainberg Y, et al. Among authors: rigo f. Nature. 2015 Dec 3;528(7580):123-6. doi: 10.1038/nature16159. Epub 2015 Nov 25. Nature. 2015. PMID: 26605526 Free PMC article.

18

The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.

d'Ydewalle C, Ramos DM, Pyles NJ, Ng SY, Gorz M, Pilato CM, Ling K, Kong L, Ward AJ, Rubin LL, Rigo F, Bennett CF, Sumner CJ. d'Ydewalle C, et al. Among authors: rigo f. Neuron. 2017 Jan 4;93(1):66-79. doi: 10.1016/j.neuron.2016.11.033. Epub 2016 Dec 22. Neuron. 2017. PMID: 28017471 Free PMC article.

19

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.

Scoles DR, Meera P, Schneider MD, Paul S, Dansithong W, Figueroa KP, Hung G, Rigo F, Bennett CF, Otis TS, Pulst SM. Scoles DR, et al. Among authors: rigo f. Nature. 2017 Apr 20;544(7650):362-366. doi: 10.1038/nature22044. Epub 2017 Apr 12. Nature. 2017. PMID: 28405024 Free PMC article.

20

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice.

Toonen LJA, Rigo F, van Attikum H, van Roon-Mom WMC. Toonen LJA, et al. Among authors: rigo f. Mol Ther Nucleic Acids. 2017 Sep 15;8:232-242. doi: 10.1016/j.omtn.2017.06.019. Epub 2017 Jun 29. Mol Ther Nucleic Acids. 2017. PMID: 28918024 Free PMC article.

21

Antisense-mediated reduction of EphA4 in the adult CNS does not improve the function of mice with amyotrophic lateral sclerosis.

Ling KK, Jackson M, Alkam D, Liu D, Allaire N, Sun C, Kiaei M, McCampbell A, Rigo F. Ling KK, et al. Among authors: rigo f. Neurobiol Dis. 2018 Jun;114:174-183. doi: 10.1016/j.nbd.2018.03.002. Epub 2018 Mar 6. Neurobiol Dis. 2018. PMID: 29518482 Free PMC article.

22

Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice.

Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, Rigo F, Bennett CF, Krainer AR. Sinha R, et al. Among authors: rigo f. Nucleic Acids Res. 2018 Jun 1;46(10):4833-4844. doi: 10.1093/nar/gky249. Nucleic Acids Res. 2018. PMID: 29672717 Free PMC article.

23

Evaluation of the effect of 2'-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice.

Prakash TP, Yu J, Kinberger GA, Low A, Jackson M, Rigo F, Swayze EE, Seth PP. Prakash TP, et al. Among authors: rigo f. Bioorg Med Chem Lett. 2018 Dec 15;28(23-24):3774-3779. doi: 10.1016/j.bmcl.2018.10.011. Epub 2018 Oct 10. Bioorg Med Chem Lett. 2018. PMID: 30342955

24

Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor.

Scharner J, Qi S, Rigo F, Bennett CF, Krainer AR. Scharner J, et al. Among authors: rigo f. Mol Ther Nucleic Acids. 2019 Jun 7;16:313-325. doi: 10.1016/j.omtn.2019.02.024. Epub 2019 Mar 13. Mol Ther Nucleic Acids. 2019. PMID: 30965276 Free PMC article.

25

Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates.

Østergaard ME, Jackson M, Low A, E Chappell A, G Lee R, Peralta RQ, Yu J, Kinberger GA, Dan A, Carty R, Tanowitz M, Anderson P, Kim TW, Fradkin L, Mullick AE, Murray S, Rigo F, Prakash TP, Bennett CF, Swayze EE, Gaus HJ, Seth PP. Østergaard ME, et al. Among authors: rigo f. Nucleic Acids Res. 2019 Jul 9;47(12):6045-6058. doi: 10.1093/nar/gkz360. Nucleic Acids Res. 2019. PMID: 31076766 Free PMC article.

26

NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice.

Torres-Benito L, Schneider S, Rombo R, Ling KK, Grysko V, Upadhyay A, Kononenko NL, Rigo F, Bennett CF, Wirth B. Torres-Benito L, et al. Among authors: rigo f. Am J Hum Genet. 2019 Jul 3;105(1):221-230. doi: 10.1016/j.ajhg.2019.05.008. Epub 2019 Jun 20. Am J Hum Genet. 2019. PMID: 31230718 Free PMC article.

27

Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides.

Scharner J, Ma WK, Zhang Q, Lin KT, Rigo F, Bennett CF, Krainer AR. Scharner J, et al. Among authors: rigo f. Nucleic Acids Res. 2020 Jan 24;48(2):802-816. doi: 10.1093/nar/gkz1132. Nucleic Acids Res. 2020. PMID: 31802121 Free PMC article.

28

Directed RNase H Cleavage of Nascent Transcripts Causes Transcription Termination.

Lai F, Damle SS, Ling KK, Rigo F. Lai F, et al. Among authors: rigo f. Mol Cell. 2020 Mar 5;77(5):1032-1043.e4. doi: 10.1016/j.molcel.2019.12.029. Epub 2020 Jan 7. Mol Cell. 2020. PMID: 31924447 Free article.

29

Systemic therapy in an RNA toxicity mouse model with an antisense oligonucleotide therapy targeting a non-CUG sequence within the DMPK 3'UTR RNA.

Yadava RS, Yu Q, Mandal M, Rigo F, Bennett CF, Mahadevan MS. Yadava RS, et al. Among authors: rigo f. Hum Mol Genet. 2020 Jun 3;29(9):1440-1453. doi: 10.1093/hmg/ddaa060. Hum Mol Genet. 2020. PMID: 32242217 Free PMC article.

30

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.

31

Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome.

Puttaraju M, Jackson M, Klein S, Shilo A, Bennett CF, Gordon L, Rigo F, Misteli T. Puttaraju M, et al. Among authors: rigo f. Nat Med. 2021 Mar;27(3):526-535. doi: 10.1038/s41591-021-01262-4. Epub 2021 Mar 11. Nat Med. 2021. PMID: 33707772 Free PMC article.

32

Modeling muscle regeneration in RNA toxicity mice.

Yadava RS, Mandal M, Giese JM, Rigo F, Bennett CF, Mahadevan MS. Yadava RS, et al. Among authors: rigo f. Hum Mol Genet. 2021 Jun 9;30(12):1111-1130. doi: 10.1093/hmg/ddab108. Hum Mol Genet. 2021. PMID: 33864373 Free PMC article.

33

Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy.

Bouwman LF, den Hamer B, van den Heuvel A, Franken M, Jackson M, Dwyer CA, Tapscott SJ, Rigo F, van der Maarel SM, de Greef JC. Bouwman LF, et al. Among authors: rigo f. Mol Ther Nucleic Acids. 2021 Sep 27;26:813-827. doi: 10.1016/j.omtn.2021.09.010. eCollection 2021 Dec 3. Mol Ther Nucleic Acids. 2021. PMID: 34729250 Free PMC article.

34

Sarm1 haploinsufficiency or low expression levels after antisense oligonucleotides delay programmed axon degeneration.

Gould SA, Gilley J, Ling K, Jafar-Nejad P, Rigo F, Coleman M. Gould SA, et al. Among authors: rigo f. Cell Rep. 2021 Dec 14;37(11):110108. doi: 10.1016/j.celrep.2021.110108. Cell Rep. 2021. PMID: 34910914 Free PMC article.

35

Difficulties translating antisense-mediated activation of Frataxin expression from cell culture to mice.

Kilikevicius A, Wang J, Shen X, Rigo F, Prakash TP, Napierala M, Corey DR. Kilikevicius A, et al. Among authors: rigo f. RNA Biol. 2022;19(1):364-372. doi: 10.1080/15476286.2022.2043650. Epub 2021 Dec 31. RNA Biol. 2022. PMID: 35289725 Free PMC article.

36

Non-invasive Imaging of Antisense Oligonucleotides in the Brain via In Vivo Click Chemistry.

Cook BE, Archbold J, Nasr K, Girmay S, Goldstein SI, Li P, Dandapani S, Genung NE, Tang SP, McClusky S, Plisson C, Afetian ME, Dwyer CA, Fazio M, Drury WJ 3rd, Rigo F, Martarello L, Kaliszczak M. Cook BE, et al. Among authors: rigo f. Mol Imaging Biol. 2022 Dec;24(6):940-949. doi: 10.1007/s11307-022-01744-y. Epub 2022 Jun 2. Mol Imaging Biol. 2022. PMID: 35655109

37

Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing.

Rigo F, Hua Y, Chun SJ, Prakash TP, Krainer AR, Bennett CF. Rigo F, et al. Nat Chem Biol. 2012 Apr 15;8(6):555-61. doi: 10.1038/nchembio.939. Nat Chem Biol. 2012. PMID: 22504300 Free PMC article.

38

Antisense-based therapy for the treatment of spinal muscular atrophy.

Rigo F, Hua Y, Krainer AR, Bennett CF. Rigo F, et al. J Cell Biol. 2012 Oct 1;199(1):21-5. doi: 10.1083/jcb.201207087. J Cell Biol. 2012. PMID: 23027901 Free PMC article.

39

Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides.

Ward AJ, Norrbom M, Chun S, Bennett CF, Rigo F. Ward AJ, et al. Among authors: rigo f. Nucleic Acids Res. 2014 May;42(9):5871-9. doi: 10.1093/nar/gku184. Epub 2014 Mar 3. Nucleic Acids Res. 2014. PMID: 24589581 Free PMC article.

40

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.

Jiang J, Zhu Q, Gendron TF, Saberi S, McAlonis-Downes M, Seelman A, Stauffer JE, Jafar-Nejad P, Drenner K, Schulte D, Chun S, Sun S, Ling SC, Myers B, Engelhardt J, Katz M, Baughn M, Platoshyn O, Marsala M, Watt A, Heyser CJ, Ard MC, De Muynck L, Daughrity LM, Swing DA, Tessarollo L, Jung CJ, Delpoux A, Utzschneider DT, Hedrick SM, de Jong PJ, Edbauer D, Van Damme P, Petrucelli L, Shaw CE, Bennett CF, Da Cruz S, Ravits J, Rigo F, Cleveland DW, Lagier-Tourenne C. Jiang J, et al. Among authors: rigo f. Neuron. 2016 May 4;90(3):535-50. doi: 10.1016/j.neuron.2016.04.006. Epub 2016 Apr 21. Neuron. 2016. PMID: 27112497 Free PMC article.

41

Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat.

Li L, Shen X, Liu Z, Norrbom M, Prakash TP, O'Reilly D, Sharma VK, Damha MJ, Watts JK, Rigo F, Corey DR. Li L, et al. Among authors: rigo f. Nucleic Acid Ther. 2018 Feb;28(1):23-33. doi: 10.1089/nat.2017.0703. Epub 2018 Jan 17. Nucleic Acid Ther. 2018. PMID: 29341839 Free PMC article.

42

Murine knockin model for progranulin-deficient frontotemporal dementia with nonsense-mediated mRNA decay.

Nguyen AD, Nguyen TA, Zhang J, Devireddy S, Zhou P, Karydas AM, Xu X, Miller BL, Rigo F, Ferguson SM, Huang EJ, Walther TC, Farese RV Jr. Nguyen AD, et al. Among authors: rigo f. Proc Natl Acad Sci U S A. 2018 Mar 20;115(12):E2849-E2858. doi: 10.1073/pnas.1722344115. Epub 2018 Mar 6. Proc Natl Acad Sci U S A. 2018. PMID: 29511098 Free PMC article.

43

Reduction of matrix metalloproteinase 9 (MMP-9) protects motor neurons from TDP-43-triggered death in rNLS8 mice.

Spiller KJ, Khan T, Dominique MA, Restrepo CR, Cotton-Samuel D, Levitan M, Jafar-Nejad P, Zhang B, Soriano A, Rigo F, Trojanowski JQ, Lee VM. Spiller KJ, et al. Among authors: rigo f. Neurobiol Dis. 2019 Apr;124:133-140. doi: 10.1016/j.nbd.2018.11.013. Epub 2018 Nov 17. Neurobiol Dis. 2019. PMID: 30458231 Free PMC article.

44

Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1.

Hu N, Antoury L, Baran TM, Mitra S, Bennett CF, Rigo F, Foster TH, Wheeler TM. Hu N, et al. Among authors: rigo f. Nat Commun. 2018 Dec 7;9(1):5227. doi: 10.1038/s41467-018-07517-y. Nat Commun. 2018. PMID: 30531949 Free PMC article.

45

Efficient electroporation of neuronal cells using synthetic oligonucleotides: identifying duplex RNA and antisense oligonucleotide activators of human frataxin expression.

Shen X, Beasley S, Putman JN, Li Y, Prakash TP, Rigo F, Napierala M, Corey DR. Shen X, et al. Among authors: rigo f. RNA. 2019 Sep;25(9):1118-1129. doi: 10.1261/rna.071290.119. Epub 2019 May 31. RNA. 2019. PMID: 31151992 Free PMC article.

46

Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome.

Lenk GM, Jafar-Nejad P, Hill SF, Huffman LD, Smolen CE, Wagnon JL, Petit H, Yu W, Ziobro J, Bhatia K, Parent J, Giger RJ, Rigo F, Meisler MH. Lenk GM, et al. Among authors: rigo f. Ann Neurol. 2020 Mar;87(3):339-346. doi: 10.1002/ana.25676. Epub 2020 Feb 6. Ann Neurol. 2020. PMID: 31943325 Free PMC article.

47

Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model.

Sheng L, Rigo F, Bennett CF, Krainer AR, Hua Y. Sheng L, et al. Among authors: rigo f. Nucleic Acids Res. 2020 Apr 6;48(6):2853-2865. doi: 10.1093/nar/gkaa126. Nucleic Acids Res. 2020. PMID: 32103257 Free PMC article.

48

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: rigo f. J Clin Invest. 2021 Dec 1;131(23):e152079. doi: 10.1172/JCI152079. J Clin Invest. 2021. PMID: 34850743 Free PMC article.

49

Modulation of Gene Expression in the Eye with Antisense Oligonucleotides.

Hu J, Gong X, Fan Y, Aguilar S, Rigo F, Prakash TP, Corey DR, Mootha VV. Hu J, et al. Among authors: rigo f. Nucleic Acid Ther. 2023 Nov 2. doi: 10.1089/nat.2023.0044. Online ahead of print. Nucleic Acid Ther. 2023. PMID: 37917066

50

Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.

Rigo F, Chun SJ, Norris DA, Hung G, Lee S, Matson J, Fey RA, Gaus H, Hua Y, Grundy JS, Krainer AR, Henry SP, Bennett CF. Rigo F, et al. J Pharmacol Exp Ther. 2014 Jul;350(1):46-55. doi: 10.1124/jpet.113.212407. Epub 2014 Apr 30. J Pharmacol Exp Ther. 2014. PMID: 24784568 Free PMC article.

51

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA.

Meng L, Ward AJ, Chun S, Bennett CF, Beaudet AL, Rigo F. Meng L, et al. Among authors: rigo f. Nature. 2015 Feb 19;518(7539):409-12. doi: 10.1038/nature13975. Epub 2014 Dec 1. Nature. 2015. PMID: 25470045 Free PMC article.

52

Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7.

Staropoli JF, Li H, Chun SJ, Allaire N, Cullen P, Thai A, Fleet CM, Hua Y, Bennett CF, Krainer AR, Kerr D, McCampbell A, Rigo F, Carulli JP. Staropoli JF, et al. Among authors: rigo f. Genomics. 2015 Apr;105(4):220-8. doi: 10.1016/j.ygeno.2015.01.007. Epub 2015 Jan 31. Genomics. 2015. PMID: 25645699 Free article.

53

Poly(A) Signal-Dependent Transcription Termination Occurs through a Conformational Change Mechanism that Does Not Require Cleavage at the Poly(A) Site.

Zhang H, Rigo F, Martinson HG. Zhang H, et al. Among authors: rigo f. Mol Cell. 2015 Aug 6;59(3):437-48. doi: 10.1016/j.molcel.2015.06.008. Epub 2015 Jul 9. Mol Cell. 2015. PMID: 26166703 Free article.

54

Knockdown of GADD34 in neonatal mutant SOD1 mice ameliorates ALS.

Ghadge GD, Sonobe Y, Camarena A, Drigotas C, Rigo F, Ling KK, Roos RP. Ghadge GD, et al. Among authors: rigo f. Neurobiol Dis. 2020 Mar;136:104702. doi: 10.1016/j.nbd.2019.104702. Epub 2019 Dec 16. Neurobiol Dis. 2020. PMID: 31837419 Free article.

55

Progress towards drug discovery for Friedreich's Ataxia: Identifying synthetic oligonucleotides that more potently activate expression of human frataxin protein.

Shen X, Wong J, Prakash TP, Rigo F, Li Y, Napierala M, Corey DR. Shen X, et al. Among authors: rigo f. Bioorg Med Chem. 2020 Jun 1;28(11):115472. doi: 10.1016/j.bmc.2020.115472. Epub 2020 Apr 5. Bioorg Med Chem. 2020. PMID: 32279920 Free PMC article.

56

Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease.

Elitt MS, Barbar L, Shick HE, Powers BE, Maeno-Hikichi Y, Madhavan M, Allan KC, Nawash BS, Gevorgyan AS, Hung S, Nevin ZS, Olsen HE, Hitomi M, Schlatzer DM, Zhao HT, Swayze A, LePage DF, Jiang W, Conlon RA, Rigo F, Tesar PJ. Elitt MS, et al. Among authors: rigo f. Nature. 2020 Sep;585(7825):397-403. doi: 10.1038/s41586-020-2494-3. Epub 2020 Jul 1. Nature. 2020. PMID: 32610343 Free PMC article.

57

Antisense Oligonucleotide-Mediated Reduction of HDAC6 Does Not Reduce Tau Pathology in P301S Tau Transgenic Mice.

Valencia A, Bieber VLR, Bajrami B, Marsh G, Hamann S, Wei R, Ling K, Rigo F, Arnold HM, Golonzhka O, Hering H. Valencia A, et al. Among authors: rigo f. Front Neurol. 2021 Jun 28;12:624051. doi: 10.3389/fneur.2021.624051. eCollection 2021. Front Neurol. 2021. PMID: 34262517 Free PMC article.

58

Astrocytic 4R tau expression drives astrocyte reactivity and dysfunction.

Ezerskiy LA, Schoch KM, Sato C, Beltcheva M, Horie K, Rigo F, Martynowicz R, Karch CM, Bateman RJ, Miller TM. Ezerskiy LA, et al. Among authors: rigo f. JCI Insight. 2022 Jan 11;7(1):e152012. doi: 10.1172/jci.insight.152012. JCI Insight. 2022. PMID: 34874917 Free PMC article.

59

Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome.

Spencer ER, Shi W, Komorowski RW, Gilbert JP, Ostrowski LM, Bird LM, Thibert R, Bao C, Molloy F, Calhoun M, Koirala S, Jafar-Nejad P, Rigo F, Kramer MA, Chu CJ. Spencer ER, et al. Among authors: rigo f. Brain Commun. 2022 Apr 26;4(3):fcac106. doi: 10.1093/braincomms/fcac106. eCollection 2022. Brain Commun. 2022. PMID: 35611307 Free PMC article.

60

Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome.

Lee D, Chen W, Kaku HN, Zhuo X, Chao ES, Soriano A, Kuncheria A, Flores S, Kim JH, Rivera A, Rigo F, Jafar-Nejad P, Beaudet AL, Caudill MS, Xue M. Lee D, et al. Among authors: rigo f. Elife. 2023 Jan 3;12:e81892. doi: 10.7554/eLife.81892. Elife. 2023. PMID: 36594817 Free PMC article.

61

Long-Term SMN- and Ncald-ASO Combinatorial Therapy in SMA Mice and NCALD-ASO Treatment in hiPSC-Derived Motor Neurons Show Protective Effects.

Muiños-Bühl A, Rombo R, Ling KK, Zilio E, Rigo F, Bennett CF, Wirth B. Muiños-Bühl A, et al. Among authors: rigo f. Int J Mol Sci. 2023 Feb 20;24(4):4198. doi: 10.3390/ijms24044198. Int J Mol Sci. 2023. PMID: 36835624 Free PMC article.

62

Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation.

Aggarwal G, Banerjee S, Jones SA, Benchaar Y, Bélanger J, Sévigny M, Smith DM, Niehoff ML, Pavlack M, de Vera IMS, Petkau TL, Leavitt BR, Ling K, Jafar-Nejad P, Rigo F, Morley JE, Farr SA, Dutchak PA, Sephton CF, Nguyen AD. Aggarwal G, et al. Among authors: rigo f. J Biol Chem. 2023 Nov 17:105475. doi: 10.1016/j.jbc.2023.105475. Online ahead of print. J Biol Chem. 2023. PMID: 37981208 Free article.

63

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM. Finkel RS, et al. Among authors: rigo f. Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7. Lancet. 2016. PMID: 27939059 Clinical Trial.

64

Disrupted neuronal maturation in Angelman syndrome-derived induced pluripotent stem cells.

Fink JJ, Robinson TM, Germain ND, Sirois CL, Bolduc KA, Ward AJ, Rigo F, Chamberlain SJ, Levine ES. Fink JJ, et al. Among authors: rigo f. Nat Commun. 2017 Apr 24;8:15038. doi: 10.1038/ncomms15038. Nat Commun. 2017. PMID: 28436452 Free PMC article.

65

CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis.

Janzen E, Mendoza-Ferreira N, Hosseinibarkooie S, Schneider S, Hupperich K, Tschanz T, Grysko V, Riessland M, Hammerschmidt M, Rigo F, Bennett CF, Kye MJ, Torres-Benito L, Wirth B. Janzen E, et al. Among authors: rigo f. Brain. 2018 Aug 1;141(8):2343-2361. doi: 10.1093/brain/awy167. Brain. 2018. PMID: 29961886 Free PMC article.

66

Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice.

Strathmann EA, Peters M, Hosseinibarkooie S, Rigo FW, Bennett CF, Zaworski PG, Chen KS, Nothnagel M, Wirth B. Strathmann EA, et al. PLoS One. 2018 Sep 6;13(9):e0203398. doi: 10.1371/journal.pone.0203398. eCollection 2018. PLoS One. 2018. PMID: 30188931 Free PMC article.

67

Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs' Endothelial Corneal Dystrophy.

Hu J, Shen X, Rigo F, Prakash TP, Mootha VV, Corey DR. Hu J, et al. Among authors: rigo f. Nucleic Acid Ther. 2019 Apr;29(2):73-81. doi: 10.1089/nat.2018.0764. Epub 2019 Jan 24. Nucleic Acid Ther. 2019. PMID: 30676271 Free PMC article.

68

Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome.

Shao Y, Sztainberg Y, Wang Q, Bajikar SS, Trostle AJ, Wan YW, Jafar-Nejad P, Rigo F, Liu Z, Tang J, Zoghbi HY. Shao Y, et al. Among authors: rigo f. Sci Transl Med. 2021 Mar 3;13(583):eaaz7785. doi: 10.1126/scitranslmed.aaz7785. Sci Transl Med. 2021. PMID: 33658357 Free PMC article.

69

Author Correction: Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome.

Puttaraju M, Jackson M, Klein S, Shilo A, Bennett CF, Gordon L, Rigo F, Misteli T. Puttaraju M, et al. Among authors: rigo f. Nat Med. 2021 Jul;27(7):1309. doi: 10.1038/s41591-021-01415-5. Nat Med. 2021. PMID: 34075250 No abstract available.

70

Acute Trem2 reduction triggers increased microglial phagocytosis, slowing amyloid deposition in mice.

Schoch KM, Ezerskiy LA, Morhaus MM, Bannon RN, Sauerbeck AD, Shabsovich M, Jafar-Nejad P, Rigo F, Miller TM. Schoch KM, et al. Among authors: rigo f. Proc Natl Acad Sci U S A. 2021 Jul 6;118(27):e2100356118. doi: 10.1073/pnas.2100356118. Proc Natl Acad Sci U S A. 2021. PMID: 34187891 Free PMC article.

71

Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.

Hagemann TL, Powers B, Lin NH, Mohamed AF, Dague KL, Hannah SC, Bachmann G, Mazur C, Rigo F, Olsen AL, Feany MB, Perng MD, Berman RF, Messing A. Hagemann TL, et al. Among authors: rigo f. Sci Transl Med. 2021 Nov 17;13(620):eabg4711. doi: 10.1126/scitranslmed.abg4711. Epub 2021 Nov 17. Sci Transl Med. 2021. PMID: 34788075 Free PMC article.

72

ASO silencing of a glycosyltransferase, Poglut1 , improves the liver phenotypes in mouse models of Alagille syndrome.

Niknejad N, Fox D, Burwinkel JL, Zarrin-Khameh N, Cho S, Soriano A, Cast AE, Lopez MF, Huppert KA, Rigo F, Huppert SS, Jafar-Nejad P, Jafar-Nejad H. Niknejad N, et al. Among authors: rigo f. Hepatology. 2023 Nov 1;78(5):1337-1351. doi: 10.1097/HEP.0000000000000380. Epub 2023 Apr 7. Hepatology. 2023. PMID: 37021797

73

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH. Passini MA, et al. Among authors: rigo f. Sci Transl Med. 2011 Mar 2;3(72):72ra18. doi: 10.1126/scitranslmed.3001777. Sci Transl Med. 2011. PMID: 21368223 Free PMC article.

74

Recognition of c9orf72 Mutant RNA by Single-Stranded Silencing RNAs.

Hu J, Rigo F, Prakash TP, Corey DR. Hu J, et al. Among authors: rigo f. Nucleic Acid Ther. 2017 Apr;27(2):87-94. doi: 10.1089/nat.2016.0655. Epub 2016 Dec 22. Nucleic Acid Ther. 2017. PMID: 28005462 Free PMC article.

75

Neurocalcin Delta Suppression Protects against Spinal Muscular Atrophy in Humans and across Species by Restoring Impaired Endocytosis.

Riessland M, Kaczmarek A, Schneider S, Swoboda KJ, Löhr H, Bradler C, Grysko V, Dimitriadi M, Hosseinibarkooie S, Torres-Benito L, Peters M, Upadhyay A, Biglari N, Kröber S, Hölker I, Garbes L, Gilissen C, Hoischen A, Nürnberg G, Nürnberg P, Walter M, Rigo F, Bennett CF, Kye MJ, Hart AC, Hammerschmidt M, Kloppenburg P, Wirth B. Riessland M, et al. Among authors: rigo f. Am J Hum Genet. 2017 Feb 2;100(2):297-315. doi: 10.1016/j.ajhg.2017.01.005. Epub 2017 Jan 26. Am J Hum Genet. 2017. PMID: 28132687 Free PMC article.

76

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: rigo f. Nature. 2017 Apr 20;544(7650):367-371. doi: 10.1038/nature22038. Epub 2017 Apr 12. Nature. 2017. PMID: 28405022 Free PMC article.

77

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.

78

Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood-brain barrier and knock down genes in the rodent CNS.

Nagata T, Dwyer CA, Yoshida-Tanaka K, Ihara K, Ohyagi M, Kaburagi H, Miyata H, Ebihara S, Yoshioka K, Ishii T, Miyata K, Miyata K, Powers B, Igari T, Yamamoto S, Arimura N, Hirabayashi H, Uchihara T, Hara RI, Wada T, Bennett CF, Seth PP, Rigo F, Yokota T. Nagata T, et al. Among authors: rigo f. Nat Biotechnol. 2021 Dec;39(12):1529-1536. doi: 10.1038/s41587-021-00972-x. Epub 2021 Aug 12. Nat Biotechnol. 2021. PMID: 34385691

79

Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides.

Wang Z, Jeon HY, Rigo F, Bennett CF, Krainer AR. Wang Z, et al. Among authors: rigo f. Open Biol. 2012 Oct;2(10):120133. doi: 10.1098/rsob.120133. Open Biol. 2012. PMID: 23155487 Free PMC article.

80

Effects on murine behavior and lifespan of selectively decreasing expression of mutant huntingtin allele by supt4h knockdown.

Cheng HM, Chern Y, Chen IH, Liu CR, Li SH, Chun SJ, Rigo F, Bennett CF, Deng N, Feng Y, Lin CS, Yan YT, Cohen SN, Cheng TH. Cheng HM, et al. Among authors: rigo f. PLoS Genet. 2015 Mar 11;11(3):e1005043. doi: 10.1371/journal.pgen.1005043. eCollection 2015 Mar. PLoS Genet. 2015. PMID: 25760041 Free PMC article.

81

Overcoming imatinib resistance conferred by the BIM deletion polymorphism in chronic myeloid leukemia with splice-switching antisense oligonucleotides.

Liu J, Bhadra M, Sinnakannu JR, Yue WL, Tan CW, Rigo F, Ong ST, Roca X. Liu J, et al. Among authors: rigo f. Oncotarget. 2017 Sep 6;8(44):77567-77585. doi: 10.18632/oncotarget.20658. eCollection 2017 Sep 29. Oncotarget. 2017. PMID: 29100409 Free PMC article.

82

Activating frataxin expression by single-stranded siRNAs targeting the GAA repeat expansion.

Shen X, Kilikevicius A, O'Reilly D, Prakash TP, Damha MJ, Rigo F, Corey DR. Shen X, et al. Among authors: rigo f. Bioorg Med Chem Lett. 2018 Sep 15;28(17):2850-2855. doi: 10.1016/j.bmcl.2018.07.033. Epub 2018 Jul 21. Bioorg Med Chem Lett. 2018. PMID: 30076049 Free PMC article.

83

ALS-associated genes in SCA2 mouse spinal cord transcriptomes.

Scoles DR, Dansithong W, Pflieger LT, Paul S, Gandelman M, Figueroa KP, Rigo F, Bennett CF, Pulst SM. Scoles DR, et al. Among authors: rigo f. Hum Mol Genet. 2020 Jun 27;29(10):1658-1672. doi: 10.1093/hmg/ddaa072. Hum Mol Genet. 2020. PMID: 32307524 Free PMC article.

84

ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth.

Ma WK, Voss DM, Scharner J, Costa ASH, Lin KT, Jeon HY, Wilkinson JE, Jackson M, Rigo F, Bennett CF, Krainer AR. Ma WK, et al. Among authors: rigo f. Cancer Res. 2022 Mar 1;82(5):900-915. doi: 10.1158/0008-5472.CAN-20-0948. Cancer Res. 2022. PMID: 34921016 Free PMC article.

85

Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2.

Hill SF, Ziobro JM, Jafar-Nejad P, Rigo F, Meisler MH. Hill SF, et al. Among authors: rigo f. Epilepsia. 2022 Oct;63(10):e125-e131. doi: 10.1111/epi.17374. Epub 2022 Aug 11. Epilepsia. 2022. PMID: 35892317 Free PMC article.

86

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Hua Y, et al. Among authors: rigo f. Genes Dev. 2010 Aug 1;24(15):1634-44. doi: 10.1101/gad.1941310. Epub 2010 Jul 12. Genes Dev. 2010. PMID: 20624852 Free PMC article.

87

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.

Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF, Krainer AR. Hua Y, et al. Among authors: rigo f. Nature. 2011 Oct 5;478(7367):123-6. doi: 10.1038/nature10485. Nature. 2011. PMID: 21979052 Free PMC article.

88

TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals.

Sahashi K, Hua Y, Ling KK, Hung G, Rigo F, Horev G, Katsuno M, Sobue G, Ko CP, Bennett CF, Krainer AR. Sahashi K, et al. Among authors: rigo f. Genes Dev. 2012 Aug 15;26(16):1874-84. doi: 10.1101/gad.197418.112. Genes Dev. 2012. PMID: 22895255 Free PMC article.

89

The long non-coding RNA Gomafu is acutely regulated in response to neuronal activation and involved in schizophrenia-associated alternative splicing.

Barry G, Briggs JA, Vanichkina DP, Poth EM, Beveridge NJ, Ratnu VS, Nayler SP, Nones K, Hu J, Bredy TW, Nakagawa S, Rigo F, Taft RJ, Cairns MJ, Blackshaw S, Wolvetang EJ, Mattick JS. Barry G, et al. Among authors: rigo f. Mol Psychiatry. 2014 Apr;19(4):486-94. doi: 10.1038/mp.2013.45. Epub 2013 Apr 30. Mol Psychiatry. 2014. PMID: 23628989

90

Antisense reduction of tau in adult mice protects against seizures.

DeVos SL, Goncharoff DK, Chen G, Kebodeaux CS, Yamada K, Stewart FR, Schuler DR, Maloney SE, Wozniak DF, Rigo F, Bennett CF, Cirrito JR, Holtzman DM, Miller TM. DeVos SL, et al. Among authors: rigo f. J Neurosci. 2013 Jul 31;33(31):12887-97. doi: 10.1523/JNEUROSCI.2107-13.2013. J Neurosci. 2013. PMID: 23904623 Free PMC article.

91

Pathological impact of SMN2 mis-splicing in adult SMA mice.

Sahashi K, Ling KK, Hua Y, Wilkinson JE, Nomakuchi T, Rigo F, Hung G, Xu D, Jiang YP, Lin RZ, Ko CP, Bennett CF, Krainer AR. Sahashi K, et al. Among authors: rigo f. EMBO Mol Med. 2013 Oct;5(10):1586-601. doi: 10.1002/emmm.201302567. Epub 2013 Sep 9. EMBO Mol Med. 2013. PMID: 24014320 Free PMC article.

92

RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.

Donnelly CJ, Zhang PW, Pham JT, Haeusler AR, Mistry NA, Vidensky S, Daley EL, Poth EM, Hoover B, Fines DM, Maragakis N, Tienari PJ, Petrucelli L, Traynor BJ, Wang J, Rigo F, Bennett CF, Blackshaw S, Sattler R, Rothstein JD. Donnelly CJ, et al. Among authors: rigo f. Neuron. 2013 Oct 16;80(2):415-28. doi: 10.1016/j.neuron.2013.10.015. Neuron. 2013. PMID: 24139042 Free PMC article.

93

Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion.

Sareen D, O'Rourke JG, Meera P, Muhammad AK, Grant S, Simpkinson M, Bell S, Carmona S, Ornelas L, Sahabian A, Gendron T, Petrucelli L, Baughn M, Ravits J, Harms MB, Rigo F, Bennett CF, Otis TS, Svendsen CN, Baloh RH. Sareen D, et al. Among authors: rigo f. Sci Transl Med. 2013 Oct 23;5(208):208ra149. doi: 10.1126/scitranslmed.3007529. Sci Transl Med. 2013. PMID: 24154603 Free PMC article.

94

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration.

Lagier-Tourenne C, Baughn M, Rigo F, Sun S, Liu P, Li HR, Jiang J, Watt AT, Chun S, Katz M, Qiu J, Sun Y, Ling SC, Zhu Q, Polymenidou M, Drenner K, Artates JW, McAlonis-Downes M, Markmiller S, Hutt KR, Pizzo DP, Cady J, Harms MB, Baloh RH, Vandenberg SR, Yeo GW, Fu XD, Bennett CF, Cleveland DW, Ravits J. Lagier-Tourenne C, et al. Among authors: rigo f. Proc Natl Acad Sci U S A. 2013 Nov 19;110(47):E4530-9. doi: 10.1073/pnas.1318835110. Epub 2013 Oct 29. Proc Natl Acad Sci U S A. 2013. PMID: 24170860 Free PMC article.

95

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models.

Hua Y, Liu YH, Sahashi K, Rigo F, Bennett CF, Krainer AR. Hua Y, et al. Among authors: rigo f. Genes Dev. 2015 Feb 1;29(3):288-97. doi: 10.1101/gad.256644.114. Epub 2015 Jan 12. Genes Dev. 2015. PMID: 25583329 Free PMC article.

96

DICER/AGO-dependent epigenetic silencing of D4Z4 repeats enhanced by exogenous siRNA suggests mechanisms and therapies for FSHD.

Lim JW, Snider L, Yao Z, Tawil R, Van Der Maarel SM, Rigo F, Bennett CF, Filippova GN, Tapscott SJ. Lim JW, et al. Among authors: rigo f. Hum Mol Genet. 2015 Sep 1;24(17):4817-28. doi: 10.1093/hmg/ddv206. Epub 2015 Jun 3. Hum Mol Genet. 2015. PMID: 26041815 Free PMC article.

97

Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay.

Nomakuchi TT, Rigo F, Aznarez I, Krainer AR. Nomakuchi TT, et al. Among authors: rigo f. Nat Biotechnol. 2016 Feb;34(2):164-6. doi: 10.1038/nbt.3427. Epub 2015 Dec 14. Nat Biotechnol. 2016. PMID: 26655495 Free PMC article.

98

Modulation of LMNA splicing as a strategy to treat prelamin A diseases.

Lee JM, Nobumori C, Tu Y, Choi C, Yang SH, Jung HJ, Vickers TA, Rigo F, Bennett CF, Young SG, Fong LG. Lee JM, et al. Among authors: rigo f. J Clin Invest. 2016 Apr 1;126(4):1592-602. doi: 10.1172/JCI85908. Epub 2016 Mar 21. J Clin Invest. 2016. PMID: 26999604 Free PMC article.

99

Increased 4R-Tau Induces Pathological Changes in a Human-Tau Mouse Model.

Schoch KM, DeVos SL, Miller RL, Chun SJ, Norrbom M, Wozniak DF, Dawson HN, Bennett CF, Rigo F, Miller TM. Schoch KM, et al. Among authors: rigo f. Neuron. 2016 Jun 1;90(5):941-7. doi: 10.1016/j.neuron.2016.04.042. Epub 2016 May 19. Neuron. 2016. PMID: 27210553 Free PMC article.

100

Distinct and shared functions of ALS-associated proteins TDP-43, FUS and TAF15 revealed by multisystem analyses.

Kapeli K, Pratt GA, Vu AQ, Hutt KR, Martinez FJ, Sundararaman B, Batra R, Freese P, Lambert NJ, Huelga SC, Chun SJ, Liang TY, Chang J, Donohue JP, Shiue L, Zhang J, Zhu H, Cambi F, Kasarskis E, Hoon S, Ares M Jr, Burge CB, Ravits J, Rigo F, Yeo GW. Kapeli K, et al. Among authors: rigo f. Nat Commun. 2016 Jul 5;7:12143. doi: 10.1038/ncomms12143. Nat Commun. 2016. PMID: 27378374 Free PMC article.

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