Murillo Bartolome, Alvaro ORCID: https://orcid.org/0000-0001-5470-9347, Alpaugh, Melanie, Peter Durairaj, Ruban Rex, Randall, Emma L., Larin, Meghan, Heraty, Laura, Aston, Alys N., Taylor, Alysha S., Mas Monteys, Alex, Stoberl, Nina, Heuchan, Aeverie E. R., Aeschlimann, Pascale, Fears, Kyle, Landles, Christian, Osborne, Georgina F., Mangin, Antoine, Bhattacharyya, Soumyasree, Metzakopian, Emmanouil, Allen, Nicholas D. ORCID: https://orcid.org/0000-0003-4009-186X, Puymirat, Jack, Bates, Gillian P., Davidson, Beverly L., Cicchetti, Francesca, Lelos, Mariah J. ORCID: https://orcid.org/0000-0001-7102-055X and Dion, Vincent ORCID: https://orcid.org/0000-0003-4953-7637
2026.
Cas9 nickase-mediated contraction of CAG/CTG repeats in vivo is accompanied by improvements in Huntington's disease pathology.
Science Translational Medicine
Item availability restricted. |
|
|
Murillo, Alvaro, Alpaugh, Melanie, Larin, Meghan, Randall, Emma L, Heraty, Laura, Durairaj, Ruban Rex, Aston, Alys N, Taylor, Alysha S, Monteys, Alex Mas, Stöberl, Nina, Heuchan, Aeverie ER, Aeschlimann, Pascale, Bhattacharyya, Soumyasree, Allen, Nicholas D ORCID: https://orcid.org/0000-0003-4009-186X, Puymirat, Jack, Davidson, Beverly L, Cicchetti, Francesca, Lelos, Mariah J ORCID: https://orcid.org/0000-0001-7102-055X and Dion, Vincent ORCID: https://orcid.org/0000-0003-4953-7637
2024.
I001 Cas9 nickase-mediated contraction of CAG repeats in Huntington’s disease.
Journal of Neurology, Neurosurgery and Psychiatry
95
(Suppl)
, pp. A141-A142.
10.1136/jnnp-2024-EHDN.283
|
|
Taylor, Alysha S., Barros, Dinis, Gobet, Nastassia, Schuepbach, Thierry, McAllister, Branduff, Aeschbach, Lorene, Randall, Emma L., Trofimenko, Evgeniya, Heuchan, Eleanor R., Barszcz, Paula, Ciosi, Marc, Morgan, Joanne, Hafford-Tear, Nathaniel J., Davidson, Alice E., Massey, Thomas H. ORCID: https://orcid.org/0000-0002-9804-2131, Monckton, Darren G., Jones, Lesley ORCID: https://orcid.org/0000-0002-3007-4612, network, REGISTRY Investigators of the European Huntington's disease, Xenarios, Ioannis and Dion, Vincent ORCID: https://orcid.org/0000-0003-4953-7637
2022.
Repeat Detector: versatile sizing of expanded tandem repeats and identification of interrupted alleles from targeted DNA sequencing.
NAR Genomics and Bioinformatics
4
(4)
, lqac089.
10.1093/nargab/lqac089
|
|
|
Murillo, Alvaro, Larin, Meghan, Randall, Emma L, Taylor, Alysha, Lelos, Mariah ORCID: https://orcid.org/0000-0001-7102-055X and Dion, Vincent ORCID: https://orcid.org/0000-0003-4953-7637
2022.
I05 CRISPR-Cas9 nickase-mediated gene editing to treat Huntington’s disease.
Journal of Neurology, Neurosurgery and Psychiatry
93
(Suppl)
, A86.
10.1136/jnnp-2022-ehdn.231
|
|
Yang, Bin, Borgeaud, Alicia C., Buřičová, Marcela, Aeschbach, Lorène, Rodríguez-Lima, Oscar, Ruiz Buendía, Gustavo A., Cinesi, Cinzia, Taylor, Alysha S., Baubec, Tuncay and Dion, Vincent ORCID: https://orcid.org/0000-0003-4953-7637
2022.
Expanded CAGCTG repeats resist gene silencing mediated by targeted epigenome editing.
Human Molecular Genetics
31
(3)
, pp. 386-398.
10.1093/hmg/ddab255
|
|
|



Up a level