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piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
Loperfido M, Jarmin S, Dastidar S, Di Matteo M, Perini I, Moore M, Nair N, Samara-Kuko E, Athanasopoulos T, Tedesco FS, Dickson G, Sampaolesi M, VandenDriessche T, Chuah MK. Loperfido M, et al. Among authors: nair n. Nucleic Acids Res. 2016 Jan 29;44(2):744-60. doi: 10.1093/nar/gkv1464. Epub 2015 Dec 17. Nucleic Acids Res. 2016. PMID: 26682797 Free PMC article.
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells.
Dastidar S, Ardui S, Singh K, Majumdar D, Nair N, Fu Y, Reyon D, Samara E, Gerli MFM, Klein AF, De Schrijver W, Tipanee J, Seneca S, Tulalamba W, Wang H, Chai YC, In't Veld P, Furling D, Tedesco FS, Vermeesch JR, Joung JK, Chuah MK, VandenDriessche T. Dastidar S, et al. Among authors: nair n. Nucleic Acids Res. 2018 Sep 19;46(16):8275-8298. doi: 10.1093/nar/gky548. Nucleic Acids Res. 2018. PMID: 29947794 Free PMC article.
Recent progress in gene therapy for hemophilia.
Chuah MK, Nair N, VandenDriessche T. Chuah MK, et al. Among authors: nair n. Hum Gene Ther. 2012 Jun;23(6):557-65. doi: 10.1089/hum.2012.088. Hum Gene Ther. 2012. PMID: 22671033 Review.
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
Chuah MK, Petrus I, De Bleser P, Le Guiner C, Gernoux G, Adjali O, Nair N, Willems J, Evens H, Rincon MY, Matrai J, Di Matteo M, Samara-Kuko E, Yan B, Acosta-Sanchez A, Meliani A, Cherel G, Blouin V, Christophe O, Moullier P, Mingozzi F, VandenDriessche T. Chuah MK, et al. Among authors: nair n. Mol Ther. 2014 Sep;22(9):1605-13. doi: 10.1038/mt.2014.114. Epub 2014 Jun 23. Mol Ther. 2014. PMID: 24954473 Free PMC article.
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.
Singh K, Evens H, Nair N, Rincón MY, Sarcar S, Samara-Kuko E, Chuah MK, VandenDriessche T. Singh K, et al. Among authors: nair n. Mol Ther. 2018 May 2;26(5):1241-1254. doi: 10.1016/j.ymthe.2018.02.023. Epub 2018 Mar 6. Mol Ther. 2018. PMID: 29599079 Free PMC article.
1,280 results