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Items: 1 to 50 of 104

1.

Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials.

McDonald CM, Sajeev G, Yao Z, McDonnell E, Elfring G, Souza M, Peltz SW, Darras BT, Shieh PB, Cox DA, Landry J, Signorovitch J; ACT DMD Study Group and the Tadalafil DMD Study Group.

Muscle Nerve. 2019 Oct 10. doi: 10.1002/mus.26736. [Epub ahead of print]

PMID:
31599456
2.

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.

Friesen WJ, Johnson B, Sierra J, Zhuo J, Vazirani P, Xue X, Tomizawa Y, Baiazitov R, Morrill C, Ren H, Babu S, Moon YC, Branstrom A, Mollin A, Hedrick J, Sheedy J, Elfring G, Weetall M, Colacino JM, Welch EM, Peltz SW.

PLoS One. 2018 Oct 25;13(10):e0206158. doi: 10.1371/journal.pone.0206158. eCollection 2018.

3.

Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties.

Cao L, Weetall M, Trotta C, Cintron K, Ma J, Kim MJ, Furia B, Romfo C, Graci JD, Li W, Du J, Sheedy J, Hedrick J, Risher N, Yeh S, Qi H, Arasu T, Hwang S, Lennox W, Kong R, Petruska J, Moon YC, Babiak J, Davis TW, Jacobson A, Almstead NG, Branstrom A, Colacino JM, Peltz SW.

Mol Cancer Ther. 2019 Jan;18(1):3-16. doi: 10.1158/1535-7163.MCT-18-0863. Epub 2018 Oct 23.

PMID:
30352802
4.

The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy.

Thangarajh M, Elfring GL, Trifillis P, McIntosh J, Peltz SW; Ataluren Phase 2b Study Group.

Neurology. 2018 Sep 25;91(13):e1215-e1219. doi: 10.1212/WNL.0000000000006245. Epub 2018 Aug 22.

5.

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.

Shieh PB, Mcintosh J, Jin F, Souza M, Elfring G, Narayanan S, Trifillis P, Peltz SW, Mcdonald CM, Darras BT; THE ACT DMD STUDY GROUP.

Muscle Nerve. 2018 Nov;58(5):639-645. doi: 10.1002/mus.26191. Epub 2018 Sep 27.

6.

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.

McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group.

Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17.

PMID:
28728956
7.

The nucleoside analog clitocine is a potent and efficacious readthrough agent.

Friesen WJ, Trotta CR, Tomizawa Y, Zhuo J, Johnson B, Sierra J, Roy B, Weetall M, Hedrick J, Sheedy J, Takasugi J, Moon YC, Babu S, Baiazitov R, Leszyk JD, Davis TW, Colacino JM, Peltz SW, Welch EM.

RNA. 2017 Apr;23(4):567-577. doi: 10.1261/rna.060236.116. Epub 2017 Jan 17.

8.

Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform.

Cao L, Weetall M, Bombard J, Qi H, Arasu T, Lennox W, Hedrick J, Sheedy J, Risher N, Brooks PC, Trifillis P, Trotta C, Moon YC, Babiak J, Almstead NG, Colacino JM, Davis TW, Peltz SW.

PLoS One. 2016 Dec 16;11(12):e0168366. doi: 10.1371/journal.pone.0168366. eCollection 2016.

9.

Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation.

Weetall M, Davis T, Elfring G, Northcutt V, Cao L, Moon YC, Riebling P, Dali M, Hirawat S, Babiak J, Colacino J, Almstead N, Spiegel R, Peltz SW.

Clin Pharmacol Drug Dev. 2016 Jul;5(4):296-305. doi: 10.1002/cpdd.240. Epub 2016 Feb 2.

10.

Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F.

Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127.

11.

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP.

Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332.

12.

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

Kerem E, Konstan MW, De Boeck K, Accurso FJ, Sermet-Gaudelus I, Wilschanski M, Elborn JS, Melotti P, Bronsveld I, Fajac I, Malfroot A, Rosenbluth DB, Walker PA, McColley SA, Knoop C, Quattrucci S, Rietschel E, Zeitlin PL, Barth J, Elfring GL, Welch EM, Branstrom A, Spiegel RJ, Peltz SW, Ajayi T, Rowe SM; Cystic Fibrosis Ataluren Study Group.

Lancet Respir Med. 2014 Jul;2(7):539-47. doi: 10.1016/S2213-2600(14)70100-6. Epub 2014 May 15.

13.

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.

Finkel RS, Flanigan KM, Wong B, Bönnemann C, Sampson J, Sweeney HL, Reha A, Northcutt VJ, Elfring G, Barth J, Peltz SW.

PLoS One. 2013 Dec 11;8(12):e81302. doi: 10.1371/journal.pone.0081302. eCollection 2013.

14.

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz SW.

Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17.

15.

Identification of PTC725, an orally bioavailable small molecule that selectively targets the hepatitis C Virus NS4B protein.

Gu Z, Graci JD, Lahser FC, Breslin JJ, Jung SP, Crona JH, McMonagle P, Xia E, Liu S, Karp G, Zhu J, Huang S, Nomeir A, Weetall M, Almstead NG, Peltz SW, Tong X, Ralston R, Colacino JM.

Antimicrob Agents Chemother. 2013 Jul;57(7):3250-61. doi: 10.1128/AAC.00527-13. Epub 2013 Apr 29.

16.

Ataluren as an agent for therapeutic nonsense suppression.

Peltz SW, Morsy M, Welch EM, Jacobson A.

Annu Rev Med. 2013;64:407-25. doi: 10.1146/annurev-med-120611-144851. Epub 2012 Nov 28. Review.

17.

High-throughput screening uncovers a compound that activates latent HIV-1 and acts cooperatively with a histone deacetylase (HDAC) inhibitor.

Micheva-Viteva S, Kobayashi Y, Edelstein LC, Pacchia AL, Lee HL, Graci JD, Breslin J, Phelan BD, Miller LK, Colacino JM, Gu Z, Ron Y, Peltz SW, Dougherty JP.

J Biol Chem. 2011 Jun 17;286(24):21083-91. doi: 10.1074/jbc.M110.195537. Epub 2011 Apr 15.

18.

Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosis.

Kerem E, Wilschanski M, Miller NL, Pugatsch T, Cohen T, Blau H, Rivlin J, Shoseyov D, Reha A, Constantine S, Ajayi T, Hirawat S, Elfring GL, Peltz SW, Miller LL.

J Cyst Fibros. 2011 May;10(3):193-200. doi: 10.1016/j.jcf.2011.02.003. Epub 2011 Apr 2.

19.

Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression.

Wang B, Yang Z, Brisson BK, Feng H, Zhang Z, Welch EM, Peltz SW, Barton ER, Brown RH Jr, Sweeney HL.

J Appl Physiol (1985). 2010 Sep;109(3):901-5. doi: 10.1152/japplphysiol.01366.2009. Epub 2010 Jun 17.

20.

Targeting post-transcriptional control for drug discovery.

Peltz SW, Welch EM, Trotta CR, Davis T, Jacobson A.

RNA Biol. 2009 Jul-Aug;6(3):329-34. Epub 2009 Jul 7. Review.

PMID:
19574739
21.

Nonsense suppression activity of PTC124 (ataluren).

Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM.

Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. doi: 10.1073/pnas.0901936106. Epub 2009 Jun 8. No abstract available.

22.

HIV type-1 latency: targeted induction of proviral reservoirs.

Graci JD, Colacino JM, Peltz SW, Dougherty JP, Gu Z.

Antivir Chem Chemother. 2009;19(5):177-87. Review.

PMID:
19483266
23.

Gunvalson and PTC Therapeutics' community outreach.

Peltz SW.

Nat Biotechnol. 2009 Feb;27(2):121-2. doi: 10.1038/nbt0209-121. No abstract available.

PMID:
19204684
24.

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model.

Du M, Liu X, Welch EM, Hirawat S, Peltz SW, Bedwell DM.

Proc Natl Acad Sci U S A. 2008 Feb 12;105(6):2064-9. doi: 10.1073/pnas.0711795105. Epub 2008 Feb 6.

25.

Mining the GEMS--a novel platform technology targeting post-transcriptional control mechanisms.

Bhattacharyya A, Trotta CR, Peltz SW.

Drug Discov Today. 2007 Jul;12(13-14):553-60. Epub 2007 Jun 28. Review.

PMID:
17631250
26.

PTC124 targets genetic disorders caused by nonsense mutations.

Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL.

Nature. 2007 May 3;447(7140):87-91. Epub 2007 Apr 22.

PMID:
17450125
27.

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.

Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL.

J Clin Pharmacol. 2007 Apr;47(4):430-44.

PMID:
17389552
28.

Cleavage of pre-tRNAs by the splicing endonuclease requires a composite active site.

Trotta CR, Paushkin SV, Patel M, Li H, Peltz SW.

Nature. 2006 May 18;441(7091):375-7.

PMID:
16710424
29.

Role for Upf2p phosphorylation in Saccharomyces cerevisiae nonsense-mediated mRNA decay.

Wang W, Cajigas IJ, Peltz SW, Wilkinson MF, González CI.

Mol Cell Biol. 2006 May;26(9):3390-400.

30.

Derepression of the Her-2 uORF is mediated by a novel post-transcriptional control mechanism in cancer cells.

Mehta A, Trotta CR, Peltz SW.

Genes Dev. 2006 Apr 15;20(8):939-53. Epub 2006 Apr 5.

31.

Human immunodeficiency virus type 1 latency model for high-throughput screening.

Micheva-Viteva S, Pacchia AL, Ron Y, Peltz SW, Dougherty JP.

Antimicrob Agents Chemother. 2005 Dec;49(12):5185-8.

32.

p38 mitogen-activated protein kinase/Hog1p regulates translation of the AU-rich-element-bearing MFA2 transcript.

Vasudevan S, Garneau N, Tu Khounh D, Peltz SW.

Mol Cell Biol. 2005 Nov;25(22):9753-63.

33.

Global analysis of Pub1p targets reveals a coordinate control of gene expression through modulation of binding and stability.

Duttagupta R, Tian B, Wilusz CJ, Khounh DT, Soteropoulos P, Ouyang M, Dougherty JP, Peltz SW.

Mol Cell Biol. 2005 Jul;25(13):5499-513.

34.

A newly discovered function for RNase L in regulating translation termination.

Le Roy F, Salehzada T, Bisbal C, Dougherty JP, Peltz SW.

Nat Struct Mol Biol. 2005 Jun;12(6):505-12. Epub 2005 May 22.

PMID:
15908960
35.

Identification of a human endonuclease complex reveals a link between tRNA splicing and pre-mRNA 3' end formation.

Paushkin SV, Patel M, Furia BS, Peltz SW, Trotta CR.

Cell. 2004 Apr 30;117(3):311-21.

36.
37.

Nuclear mRNA surveillance.

Vasudevan S, Peltz SW.

Curr Opin Cell Biol. 2003 Jun;15(3):332-7. Review.

PMID:
12787776
38.

Nonsense-mediated mRNA decay in Saccharomyces cerevisiae: a quality control mechanism that degrades transcripts harboring premature termination codons.

González CI, Wang W, Peltz SW.

Cold Spring Harb Symp Quant Biol. 2001;66:321-8. Review. No abstract available.

PMID:
12762034
39.
40.

Toward the development of a virus-cell-based assay for the discovery of novel compounds against human immunodeficiency virus type 1.

Adelson ME, Pacchia AL, Kaul M, Rando RF, Ron Y, Peltz SW, Dougherty JP.

Antimicrob Agents Chemother. 2003 Feb;47(2):501-8.

41.

Non-stop decay--a new mRNA surveillance pathway.

Vasudevan S, Peltz SW, Wilusz CJ.

Bioessays. 2002 Sep;24(9):785-8. Review.

PMID:
12210514
42.

Curbing the nonsense: the activation and regulation of mRNA surveillance.

Wilusz CJ, Wang W, Peltz SW.

Genes Dev. 2001 Nov 1;15(21):2781-5. Review. No abstract available.

43.

Poly(A)-binding proteins regulate both mRNA deadenylation and decapping in yeast cytoplasmic extracts.

Wilusz CJ, Gao M, Jones CL, Wilusz J, Peltz SW.

RNA. 2001 Oct;7(10):1416-24.

44.

Nonsense-mediated mRNA decay in Saccharomyces cerevisiae.

González CI, Bhattacharya A, Wang W, Peltz SW.

Gene. 2001 Aug 22;274(1-2):15-25. Review.

PMID:
11674994
45.

Regulated ARE-mediated mRNA decay in Saccharomyces cerevisiae.

Vasudevan S, Peltz SW.

Mol Cell. 2001 Jun;7(6):1191-200.

46.

Characterization of a general stabilizer element that blocks deadenylation-dependent mRNA decay.

Ruiz-Echevarria MJ, Munshi R, Tomback J, Kinzy TG, Peltz SW.

J Biol Chem. 2001 Aug 17;276(33):30995-1003. Epub 2001 Jun 22.

47.

The cap-to-tail guide to mRNA turnover.

Wilusz CJ, Wormington M, Peltz SW.

Nat Rev Mol Cell Biol. 2001 Apr;2(4):237-46. Review.

PMID:
11283721
48.

A novel mRNA-decapping activity in HeLa cytoplasmic extracts is regulated by AU-rich elements.

Gao M, Wilusz CJ, Peltz SW, Wilusz J.

EMBO J. 2001 Mar 1;20(5):1134-43.

49.
50.

Characterization of the biochemical properties of the human Upf1 gene product that is involved in nonsense-mediated mRNA decay.

Bhattacharya A, Czaplinski K, Trifillis P, He F, Jacobson A, Peltz SW.

RNA. 2000 Sep;6(9):1226-35.

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