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56 results

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Page 1
Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates.
Tanenhaus A, Stowe T, Young A, McLaughlin J, Aeran R, Lin IW, Li J, Hosur R, Chen M, Leedy J, Chou T, Pillay S, Vila MC, Kearney JA, Moorhead M, Belle A, Tagliatela S. Tanenhaus A, et al. Among authors: vila mc. Hum Gene Ther. 2022 Jun;33(11-12):579-597. doi: 10.1089/hum.2022.037. Hum Gene Ther. 2022. PMID: 35435735 Free PMC article.
Neuron-targeted gene therapy rescues multiple phenotypes of STXBP1-related disorders in mice and is well tolerated in nonhuman primates.
Aeran R, Tanenhaus A, Sears SMS, Moerke NJ, Miller A, Artur C, Bouhlal Y, Bove PF, Diaz de Arce AJ, Shimizu S, Le J, Place K, Hoffelt D, Amarlkhagva T, Su J, Chen M, Babineau BA, McLaughlin J, Soe M, Macdonald W, Lin IW, Bole D, Valentine KM, Hallam E, Dhanota P, Liu S, Tan SA, Zhao B, Hosur R, Vila MC, Poda S, Belle A, Tagliatela S. Aeran R, et al. Among authors: vila mc. Mol Ther. 2025 Aug 6;33(8):3822-3840. doi: 10.1016/j.ymthe.2025.05.011. Epub 2025 May 9. Mol Ther. 2025. PMID: 40349107 Free article.
Severe communication delays are independent of seizure burden and persist despite contemporary treatments in SCN1A+ Dravet syndrome: Insights from the ENVISION natural history study.
Perry MS, Scheffer IE, Sullivan J, Brunklaus A, Boronat S, Wheless JW, Laux L, Patel AD, Roberts CM, Dlugos D, Holder D, Knupp KG, Lallas M, Phillips S, Segal E, Smeyers P, Lal D, Wirrell E, Zuberi S, Brünger T, Wojnaroski M, Maru B, O'Donnell P, Morton M, James E, Vila MC, Huang N, Gofshteyn JS, Rico S. Perry MS, et al. Among authors: vila mc. Epilepsia. 2024 Feb;65(2):322-337. doi: 10.1111/epi.17850. Epub 2023 Dec 22. Epilepsia. 2024. PMID: 38049202 Free article.
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Dowling JJ, Müller-Felber W, Smith BK, Bönnemann CG, Kuntz NL, Muntoni F, Servais L, Alfano LN, Beggs AH, Bilder DA, Blaschek A, Duong T, Graham RJ, Jain M, Lawlor MW, Lee J, Coats J, Lilien C, Lowes LP, MacBean V, Neuhaus S, Noursalehi M, Pitts T, Finlay C, Christensen S, Rafferty G, Seferian AM, Tsuchiya E, James ES, Miller W, Sepulveda B, Vila MC, Prasad S, Rico S, Shieh PB; INCEPTUS investigators. Dowling JJ, et al. Among authors: vila mc. J Neuromuscul Dis. 2022;9(4):503-516. doi: 10.3233/JND-210781. J Neuromuscul Dis. 2022. PMID: 35694931 Free PMC article.
ACTH stimulates turnover of the phosphatidylinositol-glycan.
Cozza EN, Vila MC, Gomez-Sanchez CE, Farese RV. Cozza EN, et al. Among authors: vila mc. Biochem Biophys Res Commun. 1988 Dec 15;157(2):585-9. doi: 10.1016/s0006-291x(88)80290-0. Biochem Biophys Res Commun. 1988. PMID: 2849431
TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy.
Fiorillo AA, Heier CR, Novak JS, Tully CB, Brown KJ, Uaesoontrachoon K, Vila MC, Ngheim PP, Bello L, Kornegay JN, Angelini C, Partridge TA, Nagaraju K, Hoffman EP. Fiorillo AA, et al. Among authors: vila mc. Cell Rep. 2015 Sep 8;12(10):1678-90. doi: 10.1016/j.celrep.2015.07.066. Epub 2015 Aug 28. Cell Rep. 2015. PMID: 26321630 Free PMC article.
56 results