Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

81 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
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. Naryshkin NA, et al. Among authors: karp gm. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
Ratni H, Karp GM, Weetall M, Naryshkin NA, Paushkin SV, Chen KS, McCarthy KD, Qi H, Turpoff A, Woll MG, Zhang X, Zhang N, Yang T, Dakka A, Vazirani P, Zhao X, Pinard E, Green L, David-Pierson P, Tuerck D, Poirier A, Muster W, Kirchner S, Mueller L, Gerlach I, Metzger F. Ratni H, et al. Among authors: karp gm. J Med Chem. 2016 Jul 14;59(13):6086-100. doi: 10.1021/acs.jmedchem.6b00459. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299419
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.
Woll MG, Qi H, Turpoff A, Zhang N, Zhang X, Chen G, Li C, Huang S, Yang T, Moon YC, Lee CS, Choi S, Almstead NG, Naryshkin NA, Dakka A, Narasimhan J, Gabbeta V, Welch E, Zhao X, Risher N, Sheedy J, Weetall M, Karp GM. Woll MG, et al. Among authors: karp gm. J Med Chem. 2016 Jul 14;59(13):6070-85. doi: 10.1021/acs.jmedchem.6b00460. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299569
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M. Zhao X, et al. Among authors: karp g. Hum Mol Genet. 2016 May 15;25(10):1885-1899. doi: 10.1093/hmg/ddw062. Epub 2016 Feb 29. Hum Mol Genet. 2016. PMID: 26931466 Free PMC article.
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. Welch EM, et al. Among authors: karp g. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
Small molecule splicing modifiers with systemic HTT-lowering activity.
Bhattacharyya A, Trotta CR, Narasimhan J, Wiedinger KJ, Li W, Effenberger KA, Woll MG, Jani MB, Risher N, Yeh S, Cheng Y, Sydorenko N, Moon YC, Karp GM, Weetall M, Dakka A, Gabbeta V, Naryshkin NA, Graci JD, Tripodi T Jr, Southwell A, Hayden M, Colacino JM, Peltz SW. Bhattacharyya A, et al. Among authors: karp gm. Nat Commun. 2021 Dec 15;12(1):7299. doi: 10.1038/s41467-021-27157-z. Nat Commun. 2021. PMID: 34911927 Free PMC article.
Identification of benzazole compounds that induce HIV-1 transcription.
Graci JD, Michaels D, Chen G, Schiralli Lester GM, Nodder S, Weetall M, Karp GM, Gu Z, Colacino JM, Henderson AJ. Graci JD, et al. Among authors: karp gm. PLoS One. 2017 Jun 28;12(6):e0179100. doi: 10.1371/journal.pone.0179100. eCollection 2017. PLoS One. 2017. PMID: 28658263 Free PMC article.
6-(Azaindol-2-yl)pyridine-3-sulfonamides as potent and selective inhibitors targeting hepatitis C virus NS4B.
Chen G, Ren H, Zhang N, Lennox W, Turpoff A, Paget S, Li C, Almstead N, Njoroge FG, Gu Z, Graci J, Jung SP, Colacino J, Lahser F, Zhao X, Weetall M, Nomeir A, Karp GM. Chen G, et al. Among authors: karp gm. Bioorg Med Chem Lett. 2015 Feb 15;25(4):781-6. doi: 10.1016/j.bmcl.2014.12.093. Epub 2015 Jan 7. Bioorg Med Chem Lett. 2015. PMID: 25613678
81 results