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Page 1
Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder.
Voronin G, Narasimhan J, Gittens J, Sheedy J, Lipari P, Peters M, DeMarco S, Cao L, Varganov Y, Kim MJ, Pear L, Fotouh E, Sinha S, Ray B, Wu MC, Yalamanchili P, Southgate C, Pick J, Saadipour K, Jung S, Lee J, Mollin A, Welch EM, Wu Z, Weetall M. Voronin G, et al. Among authors: weetall m. Mol Ther. 2024 Oct 2;32(10):3331-3345. doi: 10.1016/j.ymthe.2024.07.012. Epub 2024 Jul 20. Mol Ther. 2024. PMID: 39033321
Phase Ib Study of Unesbulin (PTC596) Plus Dacarbazine for the Treatment of Locally Recurrent, Unresectable or Metastatic, Relapsed or Refractory Leiomyosarcoma.
Van Tine BA, Ingham MA, Attia S, Meyer CF, Baird JD, Brooks-Asplund E, D'Silva D, Kong R, Mwatha A, O'Keefe K, Weetall M, Spiegel R, Schwartz GK. Van Tine BA, et al. Among authors: weetall m. J Clin Oncol. 2024 Jul 10;42(20):2404-2414. doi: 10.1200/JCO.23.01684. Epub 2024 Apr 29. J Clin Oncol. 2024. PMID: 38684039 Free PMC article. Clinical Trial.
Tumor Microenvironment Landscapes Supporting EGFR-mutant NSCLC Are Modulated at the Single-cell Interaction Level by Unesbulin Treatment.
Maroni G, Krishnan I, Alfieri R, Maymi VA, Pandell N, Csizmadia E, Zhang J, Weetall M, Branstrom A, Braccini G, Cabrera San Millán E, Storti B, Bizzarri R, Kocher O, Daniela Sanchez Bassères DS, Welner RS, Magli MC, Merelli I, Clohessy JG, Ali A, Tenen DG, Levantini E. Maroni G, et al. Among authors: weetall m. Cancer Res Commun. 2024 Mar 26;4(3):919-937. doi: 10.1158/2767-9764.CRC-23-0161. Cancer Res Commun. 2024. PMID: 38546390 Free PMC article.
Editorial: New therapeutics for soft tissue sarcomas.
Weetall M, Rance M, Ingham M, Van Tine BA. Weetall M, et al. Front Oncol. 2023 Oct 16;13:1297215. doi: 10.3389/fonc.2023.1297215. eCollection 2023. Front Oncol. 2023. PMID: 37909024 Free PMC article. No abstract available.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: weetall m. Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w. Sci Rep. 2023. PMID: 37903840 Free PMC article.
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.
Liu G, Lipari P, Mollin A, Jung S, Teplova I, Li W, Ying L, More V, Lennox W, Yeh S, McGann E, Moon YC, Rice C, Huarte E, Gruszka B, Ray B, Goodwin E, Buckendahl P, Yurkow E, Braughton B, Narasimhan J, Welch E, Voronin G, Weetall M. Liu G, et al. Among authors: weetall m. Hum Mol Genet. 2024 Jan 20;33(3):211-223. doi: 10.1093/hmg/ddad173. Hum Mol Genet. 2024. PMID: 37819629 Free PMC article.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: weetall m. bioRxiv [Preprint]. 2023 May 24:2023.05.22.541535. doi: 10.1101/2023.05.22.541535. bioRxiv. 2023. Update in: Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w. PMID: 37293016 Free PMC article. Updated. Preprint.
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia.
Morini E, Chekuri A, Logan EM, Bolduc JM, Kirchner EG, Salani M, Krauson AJ, Narasimhan J, Gabbeta V, Grover S, Dakka A, Mollin A, Jung SP, Zhao X, Zhang N, Zhang S, Arnold M, Woll MG, Naryshkin NA, Weetall M, Slaugenhaupt SA. Morini E, et al. Among authors: weetall m. Am J Hum Genet. 2023 Mar 2;110(3):531-547. doi: 10.1016/j.ajhg.2023.01.019. Epub 2023 Feb 20. Am J Hum Genet. 2023. PMID: 36809767 Free PMC article.
80 results