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Science Translational Medicine|January 18, 2019
Clinical-grade stem cell-derived retinal pigment epithelium patch rescues retinal degeneration in rodents and pigsRuchi Sharma, Vladimir Khristov, Aaron Rising, et al.
Nature Communications|April 25, 2020
Sex chromosome evolution in parasitic nematodes of humansJeremy M Foster, Alexandra Grote, John Mattick, et al.
Biorxiv : the Preprint Server for Biology|November 26, 2025
Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman SyndromeXiaona Lu, Ying Xie, Youmei Bao, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 1, 2024
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell diseaseVarun Katta, Kiera O'Keefe, Yichao Li, et al.
Nature|May 22, 2024
Selective haematological cancer eradication with preserved haematopoiesisSimon Garaudé, Romina Marone, Rosalba Lepore, et al.
Nature|April 22, 2025
Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learningRachel A Silverstein, Nahye Kim, Ann-Sophie Kroell, et al.
Nature Genetics|July 3, 2023
Potent and uniform fetal hemoglobin induction via base editingThiyagaraj Mayuranathan, Gregory A Newby, Ruopeng Feng, et al.
Nature|June 3, 2021
Base editing of haematopoietic stem cells rescues sickle cell disease in miceGregory A Newby, Jonathan S Yen, Kaitly J Woodard, et al.
Biorxiv : the Preprint Server for Biology|April 17, 2023
Interspecies regulatory landscapes and elements revealed by novel joint systematic integration of human and mouse blood cell epigenomesGuanjue Xiang, Xi He, Belinda M Giardine, et al.
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