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44 Publications listed
Arrayed dual-gRNA CRISPR screening platform for C9orf72 repeat expansion excision in patient iPSCs
Published 2026
Brain
C9orf72
University of California, San Francisco,
Innovative Genomics Institute,
University of California, Berkeley - IGI,
Cardiff University,
Wellcome Genome Campus
10.1016/j.omta.2026.201741
Arogundade OA, Lam KJK, Brown KA, Jain T, Issagholian-Lewin PO, Huang C, Rae-Hudson T, Briseno K, Wyman SK, Krishnappa N, George CA, O'Dare K, Wilson RHC, van Eijk P, Reed SH, Giannikopoulos P, Clelland CD. Mol Ther Adv. 2026 Apr 20;34(2):201741. doi: 10.
Saturation editing of RNU4-2 reveals distinct dominant and recessive disorders
Published 2026
Brain
RNU4-2
The Genome Function Laboratory, The Francis Crick Institute,
Oxford Big Data Institute,
Centre for Human Genetics, University of Oxford,
University College London (UCL)
10.1038/s41586-026-10334-9
De Jonghe, J., Kim, H.C., Adedeji, A. et al. Saturation editing of RNU4-2 reveals distinct dominant and recessive disorders. Nature 654, 429–436 (2026).
Protocol: Design and Validation of Mirtron-Mediated RNA Interference for Gene Therapy
Published 2026
Eye
University of Oxford
DOI: 10.1007/978-1-0716-5198-8_14
Methods Mol Biol . 2026:3023:195-206. doi: 10.1007/978-1-0716-5198-8_14.
Genome-scale functional mapping of the mammalian whole brain with in vivo Perturb-seq
Published 2026
Brain
University of Oxford
https://doi.org/10.64898/2026.03.16.711480. Preprint.
bioRxiv [Preprint]. 2026 Mar 18:2026.03.16.711480. doi: 10.64898/2026.03.16.711480.
Crossing the finish line towards a disease-modifying treatment for Angelman syndrome
Published 2026
Angelman Syndrome
Brain
University of North Carolina,
University of Coimbra,
Princeton University,
Roche Innovation Center Basel,
Broad Institute of MIT and Harvard,
Erasmus MC Center of Expertise for Neurodevelopmental Disorders (ENCORE),
MPM BioImpact,
Columbia University,
Institute of Developmental and Regenerative Medicine, Department of Paediatrics, University of Oxford,
University of California San Diego and Rady Children's Hospital San Diego,
University of California, San Francisco,
University of Konstanz,
Washington University School of Medicine
10.1186/s11689-026-09681-5
Judson MC, de Almeida LP, Burdine RD, Chamberlain SJ, Deverman BE, Distel B, Ehlers MD, Jalazo E, Kushner SA, Nespeca M, Sanders SJ, et al. J Neurodev Disord. 2026 Mar 7;18(1):15. doi: 10.1186/s11689-026-09681-5. PMID: 41795090; PMCID: PMC13001298.