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Items: 23

1.

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
2.

Protein production is an early biomarker for RNA-targeted therapies.

Self WK, Schoch KM, Alex J, Barthélemy N, Bollinger JG, Sato C, Cole T, Kordasiewicz HB, Swayze E, Bateman RJ, Miller TM.

Ann Clin Transl Neurol. 2018 Oct 14;5(12):1492-1504. doi: 10.1002/acn3.657. eCollection 2018 Dec.

3.

Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.

Friedrich J, Kordasiewicz HB, O'Callaghan B, Handler HP, Wagener C, Duvick L, Swayze EE, Rainwater O, Hofstra B, Benneyworth M, Nichols-Meade T, Yang P, Chen Z, Ortiz JP, Clark HB, Öz G, Larson S, Zoghbi HY, Henzler C, Orr HT.

JCI Insight. 2018 Nov 2;3(21). pii: 123193. doi: 10.1172/jci.insight.123193.

4.

Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease.

Southwell AL, Kordasiewicz HB, Langbehn D, Skotte NH, Parsons MP, Villanueva EB, Caron NS, Østergaard ME, Anderson LM, Xie Y, Cengio LD, Findlay-Black H, Doty CN, Fitsimmons B, Swayze EE, Seth PP, Raymond LA, Frank Bennett C, Hayden MR.

Sci Transl Med. 2018 Oct 3;10(461). pii: eaar3959. doi: 10.1126/scitranslmed.aar3959.

PMID:
30282695
5.

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.

6.

Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.

McLoughlin HS, Moore LR, Chopra R, Komlo R, McKenzie M, Blumenstein KG, Zhao H, Kordasiewicz HB, Shakkottai VG, Paulson HL.

Ann Neurol. 2018 Jul;84(1):64-77. doi: 10.1002/ana.25264. Epub 2018 Aug 6.

7.

PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models.

Zhao HT, Damle S, Ikeda-Lee K, Kuntz S, Li J, Mohan A, Kim A, Hung G, Scheideler MA, Scherer SS, Svaren J, Swayze EE, Kordasiewicz HB.

J Clin Invest. 2018 Jan 2;128(1):359-368. doi: 10.1172/JCI96499. Epub 2017 Dec 4.

8.

Antisense oligonucleotides selectively suppress target RNA in nociceptive neurons of the pain system and can ameliorate mechanical pain.

Mohan A, Fitzsimmons B, Zhao HT, Jiang Y, Mazur C, Swayze EE, Kordasiewicz HB.

Pain. 2018 Jan;159(1):139-149. doi: 10.1097/j.pain.0000000000001074.

PMID:
28976422
9.

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson's Disease Mouse Model.

Zhao HT, John N, Delic V, Ikeda-Lee K, Kim A, Weihofen A, Swayze EE, Kordasiewicz HB, West AB, Volpicelli-Daley LA.

Mol Ther Nucleic Acids. 2017 Sep 15;8:508-519. doi: 10.1016/j.omtn.2017.08.002. Epub 2017 Aug 10.

10.

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models.

Moore LR, Rajpal G, Dillingham IT, Qutob M, Blumenstein KG, Gattis D, Hung G, Kordasiewicz HB, Paulson HL, McLoughlin HS.

Mol Ther Nucleic Acids. 2017 Jun 16;7:200-210. doi: 10.1016/j.omtn.2017.04.005. Epub 2017 Apr 12.

11.

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.

12.

MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.

Hoye ML, Koval ED, Wegener AJ, Hyman TS, Yang C, O'Brien DR, Miller RL, Cole T, Schoch KM, Shen T, Kunikata T, Richard JP, Gutmann DH, Maragakis NJ, Kordasiewicz HB, Dougherty JD, Miller TM.

J Neurosci. 2017 May 31;37(22):5574-5586. doi: 10.1523/JNEUROSCI.3582-16.2017. Epub 2017 Apr 17.

13.

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.

14.

A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.

Southwell AL, Skotte NH, Villanueva EB, Østergaard ME, Gu X, Kordasiewicz HB, Kay C, Cheung D, Xie Y, Waltl S, Dal Cengio L, Findlay-Black H, Doty CN, Petoukhov E, Iworima D, Slama R, Ooi J, Pouladi MA, Yang XW, Swayze EE, Seth PP, Hayden MR.

Hum Mol Genet. 2017 Mar 15;26(6):1115-1132. doi: 10.1093/hmg/ddx021.

PMID:
28104789
15.

Tcf4 Regulates Synaptic Plasticity, DNA Methylation, and Memory Function.

Kennedy AJ, Rahn EJ, Paulukaitis BS, Savell KE, Kordasiewicz HB, Wang J, Lewis JW, Posey J, Strange SK, Guzman-Karlsson MC, Phillips SE, Decker K, Motley ST, Swayze EE, Ecker DJ, Michael TP, Day JJ, Sweatt JD.

Cell Rep. 2016 Sep 6;16(10):2666-2685. doi: 10.1016/j.celrep.2016.08.004. Epub 2016 Aug 25.

16.

Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.

Keiser MS, Kordasiewicz HB, McBride JL.

Hum Mol Genet. 2016 Apr 15;25(R1):R53-64. doi: 10.1093/hmg/ddv442. Epub 2015 Oct 26. Review.

17.

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.

Southwell AL, Skotte NH, Kordasiewicz HB, Østergaard ME, Watt AT, Carroll JB, Doty CN, Villanueva EB, Petoukhov E, Vaid K, Xie Y, Freier SM, Swayze EE, Seth PP, Bennett CF, Hayden MR.

Mol Ther. 2014 Dec;22(12):2093-106. doi: 10.1038/mt.2014.153. Epub 2014 Aug 7.

18.

ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43.

Arnold ES, Ling SC, Huelga SC, Lagier-Tourenne C, Polymenidou M, Ditsworth D, Kordasiewicz HB, McAlonis-Downes M, Platoshyn O, Parone PA, Da Cruz S, Clutario KM, Swing D, Tessarollo L, Marsala M, Shaw CE, Yeo GW, Cleveland DW.

Proc Natl Acad Sci U S A. 2013 Feb 19;110(8):E736-45. doi: 10.1073/pnas.1222809110. Epub 2013 Feb 4.

19.

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

Yu D, Pendergraff H, Liu J, Kordasiewicz HB, Cleveland DW, Swayze EE, Lima WF, Crooke ST, Prakash TP, Corey DR.

Cell. 2012 Aug 31;150(5):895-908. doi: 10.1016/j.cell.2012.08.002.

20.

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

Kordasiewicz HB, Stanek LM, Wancewicz EV, Mazur C, McAlonis MM, Pytel KA, Artates JW, Weiss A, Cheng SH, Shihabuddin LS, Hung G, Bennett CF, Cleveland DW.

Neuron. 2012 Jun 21;74(6):1031-44. doi: 10.1016/j.neuron.2012.05.009.

21.

Molecular pathogenesis of spinocerebellar ataxia type 6.

Kordasiewicz HB, Gomez CM.

Neurotherapeutics. 2007 Apr;4(2):285-94. Review.

PMID:
17395139
22.

Dominant-negative suppression of Cav2.1 currents by alpha(1)2.1 truncations requires the conserved interaction domain for beta subunits.

Raike RS, Kordasiewicz HB, Thompson RM, Gomez CM.

Mol Cell Neurosci. 2007 Feb;34(2):168-77. Epub 2006 Dec 11.

23.

C-termini of P/Q-type Ca2+ channel alpha1A subunits translocate to nuclei and promote polyglutamine-mediated toxicity.

Kordasiewicz HB, Thompson RM, Clark HB, Gomez CM.

Hum Mol Genet. 2006 May 15;15(10):1587-99. Epub 2006 Apr 4.

PMID:
16595610

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