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Items: 1 to 50 of 87

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.

Getting Into the Rhythm With CRISPR.

VandenDriessche T, Chuah MK.

Circ Res. 2018 Sep 28;123(8):928-930. doi: 10.1161/CIRCRESAHA.118.313876. No abstract available.

PMID:
30355043
3.

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.

Nucleic Acids Res. 2018 Sep 19;46(16):8275-8298. doi: 10.1093/nar/gky548.

4.

Haemophilia gene therapy: From trailblazer to gamechanger.

Evens H, Chuah MK, VandenDriessche T.

Haemophilia. 2018 May;24 Suppl 6:50-59. doi: 10.1111/hae.13494. Review.

PMID:
29878653
5.

A Calsequestrin Cis-Regulatory Motif Coupled to a Cardiac Troponin T Promoter Improves Cardiac Adeno-Associated Virus Serotype 9 Transduction Specificity.

Chamberlain K, Riyad JM, Garnett T, Kohlbrenner E, Mookerjee A, Elmastour F, Benard L, Chen J, VandenDriessche T, Chuah MK, Marian AJ, Hajjar RJ, Gurha P, Weber T.

Hum Gene Ther. 2018 Aug;29(8):927-937. doi: 10.1089/hum.2017.188. Epub 2018 May 9.

PMID:
29641321
6.

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.

7.

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy.

VandenDriessche T, Chuah MK.

Mol Ther. 2018 Jan 3;26(1):14-16. doi: 10.1016/j.ymthe.2017.12.007. Epub 2017 Dec 21. No abstract available.

8.

Preclinical and clinical advances in transposon-based gene therapy.

Tipanee J, Chai YC, VandenDriessche T, Chuah MK.

Biosci Rep. 2017 Dec 5;37(6). pii: BSR20160614. doi: 10.1042/BSR20160614. Print 2017 Dec 22. Review.

9.

Transposons: Moving Forward from Preclinical Studies to Clinical Trials.

Tipanee J, VandenDriessche T, Chuah MK.

Hum Gene Ther. 2017 Nov;28(11):1087-1104. doi: 10.1089/hum.2017.128. Epub 2017 Aug 22. Review.

PMID:
28920716
10.

Hemophilia Gene Therapy: Ready for Prime Time?

VandenDriessche T, Chuah MK.

Hum Gene Ther. 2017 Nov;28(11):1013-1023. doi: 10.1089/hum.2017.116. Epub 2017 Aug 3. Review.

PMID:
28793786
11.

AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model.

Verhelle A, Nair N, Everaert I, Van Overbeke W, Supply L, Zwaenepoel O, Peleman C, Van Dorpe J, Lahoutte T, Devoogdt N, Derave W, Chuah MK, VandenDriessche T, Gettemans J.

Hum Mol Genet. 2017 Aug 1;26(15):3030. doi: 10.1093/hmg/ddx207. No abstract available.

PMID:
28605435
12.

A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice.

Merlin S, Cannizzo ES, Borroni E, Bruscaggin V, Schinco P, Tulalamba W, Chuah MK, Arruda VR, VandenDriessche T, Prat M, Valente G, Follenzi A.

Mol Ther. 2017 Aug 2;25(8):1815-1830. doi: 10.1016/j.ymthe.2017.04.029. Epub 2017 May 26.

13.

AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model.

Verhelle A, Nair N, Everaert I, Van Overbeke W, Supply L, Zwaenepoel O, Peleman C, Van Dorpe J, Lahoutte T, Devoogdt N, Derave W, Chuah MK, VandenDriessche T, Gettemans J.

Hum Mol Genet. 2017 Apr 1;26(7):1353-1364. doi: 10.1093/hmg/ddx056. Erratum in: Hum Mol Genet. 2017 Aug 1;26(15):3030.

PMID:
28334940
14.

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy.

VandenDriessche T, Chuah MK.

Mol Ther. 2016 Mar;24(3):414-6. doi: 10.1038/mt.2016.29. No abstract available.

15.

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.

16.

Corrigendum: Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.

Maffioletti SM, Gerli MF, Ragazzi M, Dastidar S, Benedetti S, Loperfido M, VandenDriessche T, Chuah MK, Tedesco FS.

Nat Protoc. 2015 Sep;10(9):1457. doi: 10.1038/nprot0915-1457d. Epub 2015 Aug 27. No abstract available.

PMID:
26313482
17.

Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation.

Rincon MY, VandenDriessche T, Chuah MK.

Cardiovasc Res. 2015 Oct 1;108(1):4-20. doi: 10.1093/cvr/cvv205. Epub 2015 Aug 3. Review.

18.

Editorial: Stem Cell-Based and Gene Therapy for Hereditary Muscle Disorders.

Chuah MK.

Curr Gene Ther. 2015;15(4):327-8. No abstract available.

PMID:
26206328
19.

Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy.

Klein AF, Dastidar S, Furling D, Chuah MK.

Curr Gene Ther. 2015;15(4):329-37. Review.

PMID:
26122101
20.

Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.

Maffioletti SM, Gerli MF, Ragazzi M, Dastidar S, Benedetti S, Loperfido M, VandenDriessche T, Chuah MK, Tedesco FS.

Nat Protoc. 2015 Jul;10(7):941-58. doi: 10.1038/nprot.2015.057. Epub 2015 Jun 4. Erratum in: Nat Protoc. 2015 Sep;10(9):1457.

PMID:
26042384
21.

Moving forward toward a cure for hemophilia B.

VandenDriessche T, Chuah MK.

Mol Ther. 2015 May;23(5):809-811. doi: 10.1038/mt.2015.56. No abstract available.

22.

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Cantore A, Ranzani M, Bartholomae CC, Volpin M, Valle PD, Sanvito F, Sergi LS, Gallina P, Benedicenti F, Bellinger D, Raymer R, Merricks E, Bellintani F, Martin S, Doglioni C, D'Angelo A, VandenDriessche T, Chuah MK, Schmidt M, Nichols T, Montini E, Naldini L.

Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405.

23.

Hitting the target without pulling the trigger.

VandenDriessche T, Chuah MK.

Mol Ther. 2015 Jan;23(1):4-6. doi: 10.1038/mt.2014.230. No abstract available.

24.

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.

25.

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].

26.

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.

27.

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.

28.

Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.

Viecelli HM, Harbottle RP, Wong SP, Schlegel A, Chuah MK, VandenDriessche T, Harding CO, Thöny B.

Hepatology. 2014 Sep;60(3):1035-43. doi: 10.1002/hep.27104. Epub 2014 Jul 29.

29.

Optimizing delivery and expression of designer nucleases for genome engineering.

Chuah MK, VandenDriessche T.

Hum Gene Ther Methods. 2013 Dec;24(6):329-32. doi: 10.1089/hgtb.2013.166.

PMID:
24328735
30.

Targeting endothelial cells by gene therapy.

Vandendriessche T, Chuah MK.

Blood. 2013 Sep 19;122(12):1993-4. doi: 10.1182/blood-2013-08-518266.

31.

Vector decoys trick the immune response.

VandenDriessche T, Chuah MK.

Sci Transl Med. 2013 Jul 17;5(194):194fs28. doi: 10.1126/scitranslmed.3006633.

PMID:
23863831
32.

Gene therapy for hemophilia.

Chuah MK, Evens H, VandenDriessche T.

J Thromb Haemost. 2013 Jun;11 Suppl 1:99-110. doi: 10.1111/jth.12215. Review.

33.

Glioblastoma: bridging the gap with gene therapy.

VandenDriessche T, Chuah MK.

Lancet Oncol. 2013 Aug;14(9):789-90. doi: 10.1016/S1470-2045(13)70342-5. Epub 2013 Jul 12. No abstract available.

PMID:
23850492
34.

Recent developments in transposon-mediated gene therapy.

Di Matteo M, Belay E, Chuah MK, Vandendriessche T.

Expert Opin Biol Ther. 2012 Jul;12(7):841-58. doi: 10.1517/14712598.2012.684875. Review.

PMID:
22679910
35.

Recent progress in gene therapy for hemophilia.

Chuah MK, Nair N, VandenDriessche T.

Hum Gene Ther. 2012 Jun;23(6):557-65. doi: 10.1089/hum.2012.088. Review.

PMID:
22671033
36.

Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.

Chuah MK, Vandendriessche T.

J Thromb Haemost. 2012 Aug;10(8):1566-9. doi: 10.1111/j.1538-7836.2012.04794.x. No abstract available.

37.

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases.

Daboussi F, Zaslavskiy M, Poirot L, Loperfido M, Gouble A, Guyot V, Leduc S, Galetto R, Grizot S, Oficjalska D, Perez C, Delacôte F, Dupuy A, Chion-Sotinel I, Le Clerre D, Lebuhotel C, Danos O, Lemaire F, Oussedik K, Cédrone F, Epinat JC, Smith J, Yáñez-Muñoz RJ, Dickson G, Popplewell L, Koo T, VandenDriessche T, Chuah MK, Duclert A, Duchateau P, Pâques F.

Nucleic Acids Res. 2012 Jul;40(13):6367-79. Epub 2012 Mar 29.

38.

PiggyBac toolbox.

Di Matteo M, Mátrai J, Belay E, Firdissa T, Vandendriessche T, Chuah MK.

Methods Mol Biol. 2012;859:241-54. doi: 10.1007/978-1-61779-603-6_14. Review.

PMID:
22367876
39.

Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B.

Vandendriessche T, Chuah MK.

Hum Gene Ther. 2012 Jan;23(1):4-6. doi: 10.1089/hum.2011.221. No abstract available.

PMID:
22149214
40.

Transposon-mediated gene transfer into adult and induced pluripotent stem cells.

Belay E, Dastidar S, VandenDriessche T, Chuah MK.

Curr Gene Ther. 2011 Oct;11(5):406-13. Review.

PMID:
21864290
41.

Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs.

Quattrocelli M, Palazzolo G, Floris G, Schöffski P, Anastasia L, Orlacchio A, Vandendriessche T, Chuah MK, Cossu G, Verfaillie C, Sampaolesi M.

J Pathol. 2011 Apr;223(5):593-603. doi: 10.1002/path.2845. Epub 2011 Feb 21.

PMID:
21341275
42.

Codon optimization of human factor VIII cDNAs leads to high-level expression.

Ward NJ, Buckley SM, Waddington SN, Vandendriessche T, Chuah MK, Nathwani AC, McIntosh J, Tuddenham EG, Kinnon C, Thrasher AJ, McVey JH.

Blood. 2011 Jan 20;117(3):798-807. doi: 10.1182/blood-2010-05-282707. Epub 2010 Nov 1.

43.

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.

44.

Preclinical and clinical progress in hemophilia gene therapy.

Mátrai J, Chuah MK, VandenDriessche T.

Curr Opin Hematol. 2010 Sep;17(5):387-92. doi: 10.1097/MOH.0b013e32833cd4bd. Review.

PMID:
20657281
45.

Comparative analysis of transposable element vector systems in human cells.

Grabundzija I, Irgang M, Mátés L, Belay E, Matrai J, Gogol-Döring A, Kawakami K, Chen W, Ruiz P, Chuah MK, VandenDriessche T, Izsvák Z, Ivics Z.

Mol Ther. 2010 Jun;18(6):1200-9. doi: 10.1038/mt.2010.47. Epub 2010 Apr 6.

46.

Recent advances in lentiviral vector development and applications.

Mátrai J, Chuah MK, VandenDriessche T.

Mol Ther. 2010 Mar;18(3):477-90. doi: 10.1038/mt.2009.319. Epub 2010 Jan 19. Review. Erratum in: Mol Ther. 2010 May;18(5):1055.

47.

Xenogeneic liver models for gene therapy.

Vandendriessche T, Chuah MK.

Hum Gene Ther. 2010 Jan;21(1):1-4. doi: 10.1089/hum.2009.1211. No abstract available.

PMID:
20043710
48.

Moving gene therapy forward with mobile DNA.

VandenDriessche T, Chuah MK.

Hum Gene Ther. 2009 Dec;20(12):1559-61. doi: 10.1089/hum.2009.1109. No abstract available.

PMID:
19919494
49.

Absence of thrombospondin-2 causes age-related dilated cardiomyopathy.

Swinnen M, Vanhoutte D, Van Almen GC, Hamdani N, Schellings MW, D'hooge J, Van der Velden J, Weaver MS, Sage EH, Bornstein P, Verheyen FK, VandenDriessche T, Chuah MK, Westermann D, Paulus WJ, Van de Werf F, Schroen B, Carmeliet P, Pinto YM, Heymans S.

Circulation. 2009 Oct 20;120(16):1585-97. doi: 10.1161/CIRCULATIONAHA.109.863266. Epub 2009 Oct 5.

PMID:
19805649
50.

Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors.

Izsvák Z, Chuah MK, Vandendriessche T, Ivics Z.

Methods. 2009 Nov;49(3):287-97. doi: 10.1016/j.ymeth.2009.07.001. Epub 2009 Jul 15. Review.

PMID:
19615447

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