Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 10

1.

Next-generation muscle-directed gene therapy by in silico vector design.

Sarcar S, Tulalamba W, Rincon MY, Tipanee J, Pham HQ, Evens H, Boon D, Samara-Kuko E, Keyaerts M, Loperfido M, Berardi E, Jarmin S, In't Veld P, Dickson G, Lahoutte T, Sampaolesi M, De Bleser P, VandenDriessche T, Chuah MK.

Nat Commun. 2019 Jan 30;10(1):492. doi: 10.1038/s41467-018-08283-7.

2.

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.

3.

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.

4.

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.

5.

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. 2014 Nov;22(11):2013. Waddingon, Simon N [corrected to Waddington, Simon N]. Erratum in: Mol Ther. 2015 Oct;23(10):1671.

6.

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.

7.

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.

8.

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.

9.

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.

10.

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

Supplemental Content

Loading ...
Support Center