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Items: 1 to 20 of 85

1.

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 Jun 27. doi: 10.1093/nar/gky548. [Epub ahead of print]

PMID:
29947794
2.

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.

PMID:
29878653
3.

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
4.

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.

5.

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.

PMID:
29274719
6.

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.

7.

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
8.

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
9.

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
10.

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.

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

PMID:
28334940
12.

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.

13.

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.

14.

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
15.

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.

16.

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
17.

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
18.

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
19.

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.

20.

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.

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