Showing 421 - 440 results of 6,431 for search '"editing"', query time: 0.06s Refine Results
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    High frequency CCR5 editing in human hematopoietic stem progenitor cells protects xenograft mice from HIV infection by Daniel T. Claiborne, Zachary Detwiler, Steffen S. Docken, Todd D. Borland, Deborah Cromer, Amanda Simkhovich, Youdiil Ophinni, Ken Okawa, Timothy Bateson, Tao Chen, Wesley Hudson, Radiana Trifonova, Miles P. Davenport, Tony W. Ho, Christian L. Boutwell, Todd M. Allen

    Published 2025-01-01
    “…Titration studies transplanting decreasing frequencies of CCR5 edited HSPCs demonstrate that <90% CCR5 editing confers decreasing protective benefit that becomes negligible between 54% and 26%. …”
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  5. 425

    Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration by Lewis E. Fry, Lauren Major, Ahmed Salman, Lucy A. McDermott, Jun Yang, Andrew J. King, Michelle E. McClements, Robert E. MacLaren

    Published 2025-02-01
    “…In mice injected with PspCas13b constructs, usherin protein was successfully restored and correctly localized to the connecting cilium following RNA editing. These results support the development of transcriptome targeting gene editing therapies for retinal disease.…”
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    Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy by Helena Escobar, Silvia Di Francescantonio, Julia Smirnova, Robin Graf, Stefanie Müthel, Andreas Marg, Alexej Zhogov, Supriya Krishna, Eric Metzler, Mina Petkova, Oliver Daumke, Ralf Kühn, Simone Spuler

    Published 2025-01-01
    “…We suggest that gene-edited primary muscle stem cells could exhibit utility, not only in treating dysferlin deficiency syndromes, but also perhaps other forms of muscular dystrophy.…”
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  9. 429

    Nelson textbook of pediatrics / International edition

    Published 2019
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    Book
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