Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice
2023 · Nature Biomedical Engineering · 127 citations
https://doi.org/10.1038/s41551-023-01026-0
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Rachel M. Levine
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Rachel M. Levine is an academic researcher from St. Jude Children's Research Hospital. The author has contributed to research in topics: Hemoglobinopathies and Related Disorders & RNA Interference and Gene Delivery & CRISPR and Genetic Engineering. The author has an h-index of 17, co-authored 28 publications.
ORCID: 0000-0002-3684-2950Papers by this author
worksPotent and uniform fetal hemoglobin induction via base editing
2023 · Nature Genetics · 69 citations
https://doi.org/10.1038/s41588-023-01434-7
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
2024 · Molecular Therapy · 13 citations
https://doi.org/10.1016/j.ymthe.2024.07.022
FLT1 and other candidate fetal haemoglobin modifying loci in sickle cell disease in African ancestries
2025 · Nature Communications · 5 citations
https://doi.org/10.1038/s41467-025-57413-5
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