8 Matching Annotations
  1. Nov 2025
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids
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    pmc.ncbi.nlm.nih.gov/articles/PMC12465149/
  3. Sep 2025
  4. www.sciencedirect.com www.sciencedirect.com
    Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
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    sciencedirect.com/science/article/pii/S2666379125004495
  5. Jun 2025
  6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    PPARÉ‘ variant V227A reduces plasma triglycerides through enhanced lipoprotein lipolysis
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    pmc.ncbi.nlm.nih.gov/articles/PMC12144439/
  7. May 2025
  8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods
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    pmc.ncbi.nlm.nih.gov/articles/PMC11997516/
  9. Sep 2024
  10. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Engineering Oncogenic Hotspot Mutations on SF3B1 via CRISPR-Directed PRECIS Mutagenesis
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    ncbi.nlm.nih.gov/pmc/articles/PMC11421219/
  11. Aug 2023
  12. 52.53.155.43 52.53.155.43
    One-step generation of tumor models by base editor multiplexing in adult stem cell-derived organoids
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    52.53.155.43/6be832dffc5b1bbf8ca7ed6df051bba72a6ab6af/s41467-023-40701-3-html.html
  13. May 2022
  14. rupress.org rupress.org
    Trapping of CDC42 C-terminal variants in the Golgi drives pyrin inflammasome hyperactivation
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    rupress.org/jem/article/219/6/e20211889/213184/Trapping-of-CDC42-C-terminal-variants-in-the-Golgi