28 Matching Annotations
  1. Nov 2025
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    A single non-coding SNP in FPGS modulates folate drug efficacy in acute lymphoblastic leukemia: data-driven exploration and experimental validation
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    pmc.ncbi.nlm.nih.gov/articles/PMC12638486/
  3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Prime editing for the investigation of aberrant splicing defect associated with a pathogenic PRPH2 variant
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    pmc.ncbi.nlm.nih.gov/articles/PMC12594906/
  4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    U2AF1 mutations rescue deleterious exon skipping induced by KRAS mutations
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    pmc.ncbi.nlm.nih.gov/articles/PMC11974705/
  5. Sep 2025
  6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Simvastatin overcomes the pPCK1‐pLDHA‐SPRINGlac axis‐mediated ferroptosis and chemo‐immunotherapy resistance in AKT‐hyperactivated intrahepatic cholangiocarcinoma
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12365544/
  7. Aug 2025
  8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Precise correction of G6PD Viangchan mutation in iPSCs by prime editing strategy
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12361394/
  9. Jul 2025
  10. www.cell.com www.cell.com
    In vivo prime editing rescues alternating hemiplegia of childhood in mice
    1
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    cell.com/cell/fulltext/S0092-8674(25)00740-8
  11. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Precise Correction of the Pde6b-L659P Mutation Causing Retinal Degeneration with Minimum Bystander Editing by Advanced Genome Editing Tools
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12220932/
  12. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Effects of heterozygous SMG1 mutations on nonsense-mediated mRNA decay in human pluripotent stem cell model
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12173119/
  13. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    In Vitro Correction of Point Mutations in the DYSF Gene Using Prime Editing
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12193300/
  14. Jun 2025
  15. febs.onlinelibrary.wiley.com febs.onlinelibrary.wiley.com
    FEBS Press
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    febs.onlinelibrary.wiley.com/doi/10.1002/1873-3468.70097
  16. May 2025
  17. www.sciencedirect.com www.sciencedirect.com
    Protocol to perform multiplexed assays of variant effect using curated loci prime editing
    1
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    sciencedirect.com/science/article/pii/S2666166725002576
  18. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel
    2
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    pmc.ncbi.nlm.nih.gov/articles/PMC11988564/
  19. Dec 2024
  20. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Prime Editor Gene Therapy and TREX1 Mosaicism in Retinal Vasculopathy with Cerebral Leukoencephalopathy
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11645301/
  21. Aug 2024
  22. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Protocol to create isogenic disease models from adult stem cell-derived organoids using next-generation CRISPR tools
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11298932/
  23. www.sciencedirect.com www.sciencedirect.com
    The FXR1 network acts as a signaling scaffold for actomyosin remodeling
    1
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    sciencedirect.com/science/article/pii/S009286742400775X
  24. Jun 2024
  25. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Multimodal framework to resolve variants of uncertain significance in TSC2
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11185720/
  26. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Precise editing of pathogenic nucleotide repeat expansions in iPSCs using paired prime editor
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11162781/
  27. May 2024
  28. www.sciencedirect.com www.sciencedirect.com
    Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells
    1
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    sciencedirect.com/science/article/pii/S2666379124002349
  29. Apr 2024
  30. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Massively parallel screen uncovers many rare 3′ UTR variants regulating mRNA abundance of cancer driver genes
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11026479/
  31. Mar 2024
  32. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Enhancing prime editor activity by directed protein evolution in yeast
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10920827/
  33. Feb 2024
  34. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Benzomorphan and non-benzomorphan agonists differentially alter sigma-1 receptor quaternary structure, as does types of cellular stress
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10774196/
  35. Jan 2024
  36. www.sciencedirect.com www.sciencedirect.com
    Prime-Editing of human ACTB in induced pluripotent stem cells to model human ACTB Loss-of-Function diseases and compensatory mechanisms
    1
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    sciencedirect.com/science/article/pii/S1873506124000023
  37. www.sciencedirect.com www.sciencedirect.com
    Kidney organoid models reveal cilium-autophagy metabolic axis as a therapeutic target for PKD both in vitro and in vivo
    1
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    sciencedirect.com/science/article/pii/S1934590923004332
  38. Dec 2023
  39. www.biorxiv.org www.biorxiv.org
    Creation of de novo cryptic splicing for ALS/FTD precision medicine
    1
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    biorxiv.org/content/10.1101/2023.11.15.565967v1
  40. Nov 2023
  41. www.sciencedirect.com www.sciencedirect.com
    Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need
    1
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    sciencedirect.com/science/article/pii/S0002929723003580
  42. Sep 2023
  43. www.nature.com www.nature.com
    CRISPR prime editing for unconstrained correction of oncogenic KRAS variants
    1
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    nature.com/articles/s42003-023-05052-1
  44. Jun 2023
  45. www.nature.com www.nature.com
    Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing
    1
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    nature.com/articles/s41467-023-39246-2