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Items: 1 to 50 of 236

1.

Treatment Adherence in Youth with First-Episode Psychosis: Impact of Family Support and Telehealth Delivery.

Alston M, Bennett CF, Rochani H.

Issues Ment Health Nurs. 2019 Aug 5:1-6. doi: 10.1080/01612840.2019.1630532. [Epub ahead of print]

PMID:
31381462
2.

Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy.

Montes J, Dunaway Young S, Mazzone ES, Pasternak A, Glanzman AM, Finkel RS, Darras BT, Muntoni F, Mercuri E, De Vivo DC, Bishop KM, Schneider E, Bennett CF, Foster R, Farwell W; CS2 and CS12 Study Groups.

Muscle Nerve. 2019 Jul 12. doi: 10.1002/mus.26633. [Epub ahead of print]

PMID:
31298747
3.

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.

Bennett CF, Krainer AR, Cleveland DW.

Annu Rev Neurosci. 2019 Jul 8;42:385-406. doi: 10.1146/annurev-neuro-070918-050501.

PMID:
31283897
4.

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.

Am J Hum Genet. 2019 Jul 3;105(1):221-230. doi: 10.1016/j.ajhg.2019.05.008. Epub 2019 Jun 20.

PMID:
31230718
5.

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.

Nucleic Acids Res. 2019 Jul 9;47(12):6045-6058. doi: 10.1093/nar/gkz360.

6.

Targeting Huntingtin Expression in Patients with Huntington's Disease.

Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM.

N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6.

PMID:
31059641
7.

Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies.

Darras BT, Chiriboga CA, Iannaccone ST, Swoboda KJ, Montes J, Mignon L, Xia S, Bennett CF, Bishop KM, Shefner JM, Green AM, Sun P, Bhan I, Gheuens S, Schneider E, Farwell W, De Vivo DC; ISIS-396443-CS2/ISIS-396443-CS12 Study Groups.

Neurology. 2019 May 21;92(21):e2492-e2506. doi: 10.1212/WNL.0000000000007527. Epub 2019 Apr 24.

8.

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.

Mol Ther Nucleic Acids. 2019 Jun 7;16:313-325. doi: 10.1016/j.omtn.2019.02.024. Epub 2019 Mar 13.

9.

RNA-Targeted Therapeutics.

Crooke ST, Witztum JL, Bennett CF, Baker BF.

Cell Metab. 2019 Feb 5;29(2):501. doi: 10.1016/j.cmet.2019.01.001. No abstract available.

PMID:
30726759
10.

Therapeutic Antisense Oligonucleotides Are Coming of Age.

Bennett CF.

Annu Rev Med. 2019 Jan 27;70:307-321. doi: 10.1146/annurev-med-041217-010829.

PMID:
30691367
11.

Selective tissue targeting of synthetic nucleic acid drugs.

Seth PP, Tanowitz M, Bennett CF.

J Clin Invest. 2019 Mar 1;129(3):915-925. doi: 10.1172/JCI125228. Epub 2019 Jan 28. Review.

12.

Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration.

Melamed Z, López-Erauskin J, Baughn MW, Zhang O, Drenner K, Sun Y, Freyermuth F, McMahon MA, Beccari MS, Artates JW, Ohkubo T, Rodriguez M, Lin N, Wu D, Bennett CF, Rigo F, Da Cruz S, Ravits J, Lagier-Tourenne C, Cleveland DW.

Nat Neurosci. 2019 Feb;22(2):180-190. doi: 10.1038/s41593-018-0293-z. Epub 2019 Jan 14.

13.

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.

Nat Commun. 2018 Dec 7;9(1):5227. doi: 10.1038/s41467-018-07517-y.

14.

Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.

Niu C, Prakash TP, Kim A, Quach JL, Huryn LA, Yang Y, Lopez E, Jazayeri A, Hung G, Sopher BL, Brooks BP, Swayze EE, Bennett CF, La Spada AR.

Sci Transl Med. 2018 Oct 31;10(465). pii: eaap8677. doi: 10.1126/scitranslmed.aap8677.

PMID:
30381411
15.

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.

PLoS One. 2018 Sep 6;13(9):e0203398. doi: 10.1371/journal.pone.0203398. eCollection 2018.

16.

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.

McCampbell A, Cole T, Wegener AJ, Tomassy GS, Setnicka A, Farley BJ, Schoch KM, Hoye ML, Shabsovich M, Sun L, Luo Y, Zhang M, Comfort N, Wang B, Amacker J, Thankamony S, Salzman DW, Cudkowicz M, Graham DL, Bennett CF, Kordasiewicz HB, Swayze EE, Miller TM.

J Clin Invest. 2018 Aug 1;128(8):3558-3567. doi: 10.1172/JCI99081. Epub 2018 Jul 16.

17.

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.

Brain. 2018 Aug 1;141(8):2343-2361. doi: 10.1093/brain/awy167.

18.

Translating Antisense Technology into a Treatment for Huntington's Disease.

Lane RM, Smith A, Baumann T, Gleichmann M, Norris D, Bennett CF, Kordasiewicz H.

Methods Mol Biol. 2018;1780:497-523. doi: 10.1007/978-1-4939-7825-0_23. Review.

PMID:
29856033
19.

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.

Nucleic Acids Res. 2018 Jun 1;46(10):4833-4844. doi: 10.1093/nar/gky249.

20.

Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.

McMahon MA, Prakash TP, Cleveland DW, Bennett CF, Rahdar M.

Mol Ther. 2018 May 2;26(5):1228-1240. doi: 10.1016/j.ymthe.2018.02.031. Epub 2018 Mar 6.

21.

RNA-Targeted Therapeutics.

Crooke ST, Witztum JL, Bennett CF, Baker BF.

Cell Metab. 2018 Apr 3;27(4):714-739. doi: 10.1016/j.cmet.2018.03.004. Review. Erratum in: Cell Metab. 2019 Feb 5;29(2):501.

22.

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.

Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group.

N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.

23.

Downregulation of Survivin contributes to cell-cycle arrest during postnatal cardiac development in a severe spinal muscular atrophy mouse model.

Sheng L, Wan B, Feng P, Sun J, Rigo F, Bennett CF, Akerman M, Krainer AR, Hua Y.

Hum Mol Genet. 2018 Feb 1;27(3):486-498. doi: 10.1093/hmg/ddx418.

24.

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group.

N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.

25.

Education for WIC Peer Counselors About Breastfeeding the Late Preterm Infant.

Bennett CF, Galloway C, Grassley JS.

J Nutr Educ Behav. 2018 Feb;50(2):198-202.e1. doi: 10.1016/j.jneb.2017.05.364. Epub 2017 Aug 14.

PMID:
28818488
26.

A Community Partnership to Support Breastfeeding Mothers of Late Preterm Infants.

Bennett CF, Grassley JS.

Nurs Womens Health. 2017 Aug - Sep;21(4):274-282. doi: 10.1016/j.nwh.2017.06.004.

PMID:
28784208
27.

Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice.

Jauvin D, Chrétien J, Pandey SK, Martineau L, Revillod L, Bassez G, Lachon A, MacLeod AR, Gourdon G, Wheeler TM, Thornton CA, Bennett CF, Puymirat J.

Mol Ther Nucleic Acids. 2017 Jun 16;7:465-474. doi: 10.1016/j.omtn.2017.05.007. Epub 2017 May 17.

28.

The Distinct and Cooperative Roles of Toll-Like Receptor 9 and Receptor for Advanced Glycation End Products in Modulating In Vivo Inflammatory Responses to Select CpG and Non-CpG Oligonucleotides.

Paz S, Hsiao J, Cauntay P, Soriano A, Bai L, Machemer T, Xiao X, Guo S, Hung G, Younis H, Bennett CF, Henry S, Yun TJ, Burel S.

Nucleic Acid Ther. 2017 Oct;27(5):272-284. doi: 10.1089/nat.2017.0668. Epub 2017 Jun 12.

PMID:
28605247
29.

Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.HttQ111/+ mouse model of Huntington's disease.

Coffey SR, Bragg RM, Minnig S, Ament SA, Cantle JP, Glickenhaus A, Shelnut D, Carrillo JM, Shuttleworth DD, Rodier JA, Noguchi K, Bennett CF, Price ND, Kordasiewicz HB, Carroll JB.

PLoS One. 2017 Apr 28;12(4):e0175968. doi: 10.1371/journal.pone.0175968. eCollection 2017.

30.

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.

Nature. 2017 Apr 20;544(7650):362-366. doi: 10.1038/nature22044. Epub 2017 Apr 12.

31.

Transaldolase inhibition impairs mitochondrial respiration and induces a starvation-like longevity response in Caenorhabditis elegans.

Bennett CF, Kwon JJ, Chen C, Russell J, Acosta K, Burnaevskiy N, Crane MM, Bitto A, Vander Wende H, Simko M, Pineda V, Rossner R, Wasko BM, Choi H, Chen S, Park S, Jafari G, Sands B, Perez Olsen C, Mendenhall AR, Morgan PG, Kaeberlein M.

PLoS Genet. 2017 Mar 29;13(3):e1006695. doi: 10.1371/journal.pgen.1006695. eCollection 2017 Mar.

32.

SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage.

Jangi M, Fleet C, Cullen P, Gupta SV, Mekhoubad S, Chiao E, Allaire N, Bennett CF, Rigo F, Krainer AR, Hurt JA, Carulli JP, Staropoli JF.

Proc Natl Acad Sci U S A. 2017 Mar 21;114(12):E2347-E2356. doi: 10.1073/pnas.1613181114. Epub 2017 Mar 7.

33.

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.

Am J Hum Genet. 2017 Feb 2;100(2):297-315. doi: 10.1016/j.ajhg.2017.01.005. Epub 2017 Jan 26.

34.

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.

DeVos SL, Miller RL, Schoch KM, Holmes BB, Kebodeaux CS, Wegener AJ, Chen G, Shen T, Tran H, Nichols B, Zanardi TA, Kordasiewicz HB, Swayze EE, Bennett CF, Diamond MI, Miller TM.

Sci Transl Med. 2017 Jan 25;9(374). pii: eaag0481. doi: 10.1126/scitranslmed.aag0481.

35.

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.

Neuron. 2017 Jan 4;93(1):66-79. doi: 10.1016/j.neuron.2016.11.033. Epub 2016 Dec 22.

36.

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.

Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7.

PMID:
27939059
37.

Protein-RNA Networks Regulated by Normal and ALS-Associated Mutant HNRNPA2B1 in the Nervous System.

Martinez FJ, Pratt GA, Van Nostrand EL, Batra R, Huelga SC, Kapeli K, Freese P, Chun SJ, Ling K, Gelboin-Burkhart C, Fijany L, Wang HC, Nussbacher JK, Broski SM, Kim HJ, Lardelli R, Sundararaman B, Donohue JP, Javaherian A, Lykke-Andersen J, Finkbeiner S, Bennett CF, Ares M Jr, Burge CB, Taylor JP, Rigo F, Yeo GW.

Neuron. 2016 Nov 23;92(4):780-795. doi: 10.1016/j.neuron.2016.09.050. Epub 2016 Oct 20.

38.

Pharmacology of Antisense Drugs.

Bennett CF, Baker BF, Pham N, Swayze E, Geary RS.

Annu Rev Pharmacol Toxicol. 2017 Jan 6;57:81-105. doi: 10.1146/annurev-pharmtox-010716-104846. Epub 2016 Oct 10. Review.

PMID:
27732800
39.

Mammary Tumor-Associated RNAs Impact Tumor Cell Proliferation, Invasion, and Migration.

Diermeier SD, Chang KC, Freier SM, Song J, El Demerdash O, Krasnitz A, Rigo F, Bennett CF, Spector DL.

Cell Rep. 2016 Sep 27;17(1):261-274. doi: 10.1016/j.celrep.2016.08.081.

40.

Dmpk gene deletion or antisense knockdown does not compromise cardiac or skeletal muscle function in mice.

Carrell ST, Carrell EM, Auerbach D, Pandey SK, Bennett CF, Dirksen RT, Thornton CA.

Hum Mol Genet. 2016 Oct 1;25(19):4328-4338. doi: 10.1093/hmg/ddw266. Epub 2016 Aug 13.

41.

The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype.

Hosseinibarkooie S, Peters M, Torres-Benito L, Rastetter RH, Hupperich K, Hoffmann A, Mendoza-Ferreira N, Kaczmarek A, Janzen E, Milbradt J, Lamkemeyer T, Rigo F, Bennett CF, Guschlbauer C, Büschges A, Hammerschmidt M, Riessland M, Kye MJ, Clemen CS, Wirth B.

Am J Hum Genet. 2016 Sep 1;99(3):647-665. doi: 10.1016/j.ajhg.2016.07.014. Epub 2016 Aug 4.

42.

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.

Neuron. 2016 Jun 1;90(5):941-7. doi: 10.1016/j.neuron.2016.04.042. Epub 2016 May 19.

43.

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.

Neuron. 2016 May 4;90(3):535-50. doi: 10.1016/j.neuron.2016.04.006. Epub 2016 Apr 21.

44.

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.

J Clin Invest. 2016 Apr 1;126(4):1592-602. doi: 10.1172/JCI85908. Epub 2016 Mar 21.

45.

Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.

Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Montes J, De Vivo DC, Norris DA, Bennett CF, Bishop KM.

Neurology. 2016 Mar 8;86(10):890-7. doi: 10.1212/WNL.0000000000002445. Epub 2016 Feb 10.

46.

Deletion or Inhibition of the Oxygen Sensor PHD1 Protects against Ischemic Stroke via Reprogramming of Neuronal Metabolism.

Quaegebeur A, Segura I, Schmieder R, Verdegem D, Decimo I, Bifari F, Dresselaers T, Eelen G, Ghosh D, Davidson SM, Schoors S, Broekaert D, Cruys B, Govaerts K, De Legher C, Bouché A, Schoonjans L, Ramer MS, Hung G, Bossaert G, Cleveland DW, Himmelreich U, Voets T, Lemmens R, Bennett CF, Robberecht W, De Bock K, Dewerchin M, Ghesquière B, Fendt SM, Carmeliet P.

Cell Metab. 2016 Feb 9;23(2):280-91. doi: 10.1016/j.cmet.2015.12.007. Epub 2016 Jan 7.

47.

Synthetic CRISPR RNA-Cas9-guided genome editing in human cells.

Rahdar M, McMahon MA, Prakash TP, Swayze EE, Bennett CF, Cleveland DW.

Proc Natl Acad Sci U S A. 2015 Dec 22;112(51):E7110-7. doi: 10.1073/pnas.1520883112. Epub 2015 Nov 16.

48.

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.

Burel SA, Hart CE, Cauntay P, Hsiao J, Machemer T, Katz M, Watt A, Bui HH, Younis H, Sabripour M, Freier SM, Hung G, Dan A, Prakash TP, Seth PP, Swayze EE, Bennett CF, Crooke ST, Henry SP.

Nucleic Acids Res. 2016 Mar 18;44(5):2093-109. doi: 10.1093/nar/gkv1210. Epub 2015 Nov 8.

49.

Biochemical Genetic Pathways that Modulate Aging in Multiple Species.

Bitto A, Wang AM, Bennett CF, Kaeberlein M.

Cold Spring Harb Perspect Med. 2015 Nov 2;5(11). pii: a025114. doi: 10.1101/cshperspect.a025114. Review.

50.

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.

Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5863-72. doi: 10.1073/pnas.1509758112. Epub 2015 Oct 12.

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