1. 2018
  2. 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., Shivsankaran Nair, N. N., Fu, Y., Reyon, D., Samara, E., Gerbil, M. FM., Klein, A. F., De Schrijver, W., Tipanee, J., Seneca, S., Tulalamba, W., Wang, H., Chai, Y. C., In 'T Veld, P., Furling, D., Tedesco, F. S., Vermeesch, J. R. & 3 others, Joung, J. K., Chuah, M. & VandenDriessche, T., 19 Sep 2018, In : Nucleic Acids Research. 46, 16, p. 8275-8298 24 p.

    Research output: Contribution to journalArticle

  3. Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

    Singh, K., Evens, H., Nair, N., Rincón, M. Y., Sarcar, S., Samara-Kuko, E., Chuah, M. K. & VandenDriessche, T., 2 May 2018, In : Molecular Therapy. 26, 5, p. 1241-1254 14 p.

    Research output: Contribution to journalArticle

  4. 2017
  5. CRISPR/Cas9-mediated editing for dominant genetic disorders: efficient excision of trinucleotide repeat expansion in myotonic dystrophy

    Dastidar, S., Ardui, S., Singh, K., Nair, N., Fu, Y., Reyon, D., Samara, E., Gerli, M. F. M., Klein, A. F., DeSchrijver, W., Majumdar, D., Tipanee, J., Seneca, S., Tulalamba, W., Wang, H., Chai, Y. C., In't Veld, P., Furling, D., Tedesco, F. S., Vermeesch, J. R. & 3 others, Joung, J. K., Chuah, M. K. & VandenDriessche, T., Dec 2017, In : Human Gene Therapy. 28, 12, p. A7-A7

    Research output: Contribution to journalMeeting abstract (Journal)

  6. CRISPR/Cas9-Mediated Editing of Trinucleotide Repeat Expansion in Myotonic Dystrophy

    Dastidar, S., Singh, K., Nair, N., Fu, Y., Reyon, D., Samara, E., Gerli, M. F. M., Klein, A. F., Jans, T., Tipanee, J., Seneca, S., Tulalamba, W., Wang, H., Vermeesch, J. R., Chai, Y. C., Veld, P. I., Furling, D., Tadesco, F. S., Joung, J. K., Chuah, M. K. L. & 1 others, VandenDriessche, T. C., May 2017, In : Molecular Therapy. 25, 5, p. 85-85 1 p.

    Research output: Contribution to journalMeeting abstract (Journal)

  7. 2015
  8. Genome-Wide Computational Analysis Reveals Cardiomyocyte-Specific Transcriptional CIS-Regulatory Motifs that Enable Efficient Cardiac Gene Therapy.

    Rincon Acelas, M., Sarcar, S., Danso-Abeam, D., Keyaerts, M., Matrai, J., Samara, E., Acosta-Sanchez, A., Athanasopoulos, T., Dickson, G., Lahoutte, T., De Bleser, P., VandenDriessche, T. & Chuah, M., 2015, In : Mol Ther. 23, January, p. 43-52 10 p.

    Research output: Contribution to journalArticle

  9. 2014
  10. Hyperactive PIGGYBAC Transposons for Sustained and Robust Liver-targeted Gene Therapy.

    Di Matteo, M., Samara, E., Ward, N., Mcvey, J., Chuah, M. & VandenDriessche, T., 2014, In : Mol Ther. 22, September, p. 1614-1624 11 p.

    Research output: Contribution to journalArticle

  11. Liver-Specific Transcriptional Modules Identified by Genome-Wide in Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates.

    Chuah, M., Petrus, I., De Bleser, P., Le Guiner, C., Gernoux, G., Adjali, O., Shivsankaran Nair, N. N., Willems, J., Evens, H., Rincon, M. Y., Matrai, J., Di Matteo, M., Samara, E., Yan, B., Acosta-Sanchez, A., Meliani, A., Cherel, G., Blouin, V., Christophe, O., Moulier, P. & 2 others, Mingozzi, F. & Vandendriessche, T., 2014, In : Mol Ther. 22, September, p. 1605-1613 9 p.

    Research output: Contribution to journalArticle

ID: 68772