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1982 1
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1993 3
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1995 3
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1999 3
2001 2
2004 1
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53 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.
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.
Effects of Chronic, Maximal Phosphorodiamidate Morpholino Oligomer (PMO) Dosing on Muscle Function and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy.
Benny Klimek ME, Vila MC, Edwards K, Boehler J, Novak J, Zhang A, Van der Meulen J, Tatum K, Quinn J, Fiorillo A, Burki U, Straub V, Lu QL, Hathout Y, van Den Anker J, Partridge TA, Morales M, Hoffman E, Nagaraju K. Benny Klimek ME, et al. Among authors: vila mc. J Neuromuscul Dis. 2021;8(s2):S369-S381. doi: 10.3233/JND-210701. J Neuromuscul Dis. 2021. PMID: 34569970
Elevated serum levels of interleukin-6 in endemic Burkitt lymphoma in Ghana.
Aka P, Emmanuel B, Vila MC, Jariwala A, Nkrumah F, Periago MV, Neequaye J, Kiruthu C, Levine PH, Biggar RJ, Bhatia K, Bethony JM, Mbulaiteye SM. Aka P, et al. Among authors: vila mc. Hematol Oncol. 2014 Dec;32(4):218-20. doi: 10.1002/hon.2121. Epub 2014 Jan 6. Hematol Oncol. 2014. PMID: 24493366 Free PMC article. No abstract available.
Morpholino-induced exon skipping stimulates cell-mediated and humoral responses to dystrophin in mdx mice.
Vila MC, Novak JS, Benny Klimek M, Li N, Morales M, Fritz AG, Edwards K, Boehler JF, Hogarth MW, Kinder TB, Zhang A, Mazala D, Fiorillo AA, Douglas B, Chen YW, van den Anker J, Lu QL, Hathout Y, Hoffman EP, Partridge TA, Nagaraju K. Vila MC, et al. J Pathol. 2019 Jul;248(3):339-351. doi: 10.1002/path.5263. Epub 2019 Apr 16. J Pathol. 2019. PMID: 30883742 Free PMC article.
53 results