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Items: 9

1.

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.

Mol Ther. 2018 May 2;26(5):1241-1254. doi: 10.1016/j.ymthe.2018.02.023. Epub 2018 Mar 6.

2.

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.

Nucleic Acids Res. 2016 Jan 29;44(2):744-60. doi: 10.1093/nar/gkv1464. Epub 2015 Dec 17.

3.

Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy.

Rincon MY, Sarcar S, Danso-Abeam D, Keyaerts M, Matrai J, Samara-Kuko E, Acosta-Sanchez A, Athanasopoulos T, Dickson G, Lahoutte T, De Bleser P, VandenDriessche T, Chuah MK.

Mol Ther. 2015 Jan;23(1):43-52. doi: 10.1038/mt.2014.178. Epub 2014 Sep 8.

4.

Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.

Di Matteo M, Samara-Kuko E, Ward NJ, Waddington SN, McVey JH, Chuah MK, VandenDriessche T.

Mol Ther. 2014 Sep;22(9):1614-24. doi: 10.1038/mt.2014.131. Epub 2014 Jul 18. Erratum in: Mol Ther. 2015 Oct;23(10):1671. Mol Ther. 2014 Nov;22(11):2013. Waddingon, Simon N [corrected to Waddington, Simon N].

5.

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.

Mol Ther. 2014 Sep;22(9):1605-13. doi: 10.1038/mt.2014.114. Epub 2014 Jun 23.

6.

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.

Nair N, Rincon MY, Evens H, Sarcar S, Dastidar S, Samara-Kuko E, Ghandeharian O, Man Viecelli H, Thöny B, De Bleser P, VandenDriessche T, Chuah MK.

Blood. 2014 May 15;123(20):3195-9. doi: 10.1182/blood-2013-10-534032. Epub 2014 Mar 17. Erratum in: Blood. 2015 Mar 19;125(12):2007. Dosage error in article text.

7.

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk.

Mátrai J, Cantore A, Bartholomae CC, Annoni A, Wang W, Acosta-Sanchez A, Samara-Kuko E, De Waele L, Ma L, Genovese P, Damo M, Arens A, Goudy K, Nichols TC, von Kalle C, L Chuah MK, Roncarolo MG, Schmidt M, Vandendriessche T, Naldini L.

Hepatology. 2011 May;53(5):1696-707. doi: 10.1002/hep.24230.

8.

Novel hyperactive transposons for genetic modification of induced pluripotent and adult stem cells: a nonviral paradigm for coaxed differentiation.

Belay E, Mátrai J, Acosta-Sanchez A, Ma L, Quattrocelli M, Mátés L, Sancho-Bru P, Geraerts M, Yan B, Vermeesch J, Rincón MY, Samara-Kuko E, Ivics Z, Verfaillie C, Sampaolesi M, Izsvák Z, Vandendriessche T, Chuah MK.

Stem Cells. 2010 Oct;28(10):1760-71. doi: 10.1002/stem.501.

9.

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates.

Mátés L, Chuah MK, Belay E, Jerchow B, Manoj N, Acosta-Sanchez A, Grzela DP, Schmitt A, Becker K, Matrai J, Ma L, Samara-Kuko E, Gysemans C, Pryputniewicz D, Miskey C, Fletcher B, VandenDriessche T, Ivics Z, Izsvák Z.

Nat Genet. 2009 Jun;41(6):753-61. doi: 10.1038/ng.343. Epub 2009 May 3.

PMID:
19412179

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