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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.
Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc-/- mice.
Levy C, Fusil F, Amirache F, Costa C, Girard-Gagnepain A, Negre D, Bernadin O, Garaulet G, Rodriguez A, Nair N, Vandendriessche T, Chuah M, Cosset FL, Verhoeyen E. Levy C, et al. Among authors: nair n. J Thromb Haemost. 2016 Dec;14(12):2478-2492. doi: 10.1111/jth.13520. Epub 2016 Nov 8. J Thromb Haemost. 2016. PMID: 27685947 Free article.
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.
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.
1,278 results