21 Matching Annotations
  1. Last 7 days
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Ultra-sensitive profiling of CRISPR-Cas off-target effects with Tracking-seq2
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    pmc.ncbi.nlm.nih.gov/articles/PMC13558771/
  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
    The ubiquitin ligase HUWE1 enhances WNT signaling by antagonizing destruction complex-mediated β-catenin degradation and through a mechanism independent of changes in β-catenin abundance
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    pmc.ncbi.nlm.nih.gov/articles/PMC12148233/
  7. Apr 2025
  8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Precision genome editing and in-cell measurements of oxidative DNA damage repair enable functional and mechanistic characterization of cancer-associated MUTYH variants
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    pmc.ncbi.nlm.nih.gov/articles/PMC11952967/
  9. Mar 2025
  10. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Massively parallel interrogation of human functional variants modulating cancer immunosurveillance
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    pmc.ncbi.nlm.nih.gov/articles/PMC11920242/
  11. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Bi-allelic KICS2 mutations impair KICSTOR complex-mediated mTORC1 regulation, causing intellectual disability and epilepsy
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    pmc.ncbi.nlm.nih.gov/articles/PMC11866974/
  12. Feb 2025
  13. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Optimal SpCas9- and SaCas9-mediated gene editing by enhancing gRNA transcript levels through scaffold poly-T tract reduction
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    pmc.ncbi.nlm.nih.gov/articles/PMC11823040/
  14. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Enhanced genome editing with a Streptococcus equinus Cas9
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    pmc.ncbi.nlm.nih.gov/articles/PMC11806022/
  15. Dec 2024
  16. www.sciencedirect.com www.sciencedirect.com
    Mapping functional elements of the DNA damage response through base editor screens
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    sciencedirect.com/science/article/pii/S2211124724013986
  17. Sep 2024
  18. www.sciencedirect.com www.sciencedirect.com
    Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells
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    sciencedirect.com/science/article/pii/S1096719224004529
  19. Jul 2024
  20. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells
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    ncbi.nlm.nih.gov/pmc/articles/PMC11214518/
  21. Jun 2024
  22. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Susceptibility to innate immune activation in genetically mediated myocarditis
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    ncbi.nlm.nih.gov/pmc/articles/PMC11213508/
  23. May 2024
  24. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    ERLIN1/2 scaffolds bridge TMUB1 and RNF170 and restrict cholesterol esterification to regulate the secretory pathway
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11116810/
  25. Apr 2024
  26. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    A de novo dominant-negative variant is associated with OTULIN-related autoinflammatory syndrome
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    ncbi.nlm.nih.gov/pmc/articles/PMC11040501/
  27. Mar 2024
  28. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Primate Model Carrying LMNA Mutation Develops Dilated Cardiomyopathy
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    ncbi.nlm.nih.gov/pmc/articles/PMC10978409/
  29. www.sciencedirect.com www.sciencedirect.com
    The Y49H cytochrome c variant enhances megakaryocytic maturation of K-562 cells
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    sciencedirect.com/science/article/pii/S0925443924001236
  30. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    The ubiquitin ligase HUWE1 enhances WNT signaling by antagonizing destruction complex-mediated β-catenin degradation and through a mechanism independent of β-catenin stability
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10896346/
  31. Sep 2023
  32. www.sciencedirect.com www.sciencedirect.com
    Base editing corrects the common Salla disease SLC17A5 c.115C>T variant
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    sciencedirect.com/science/article/pii/S2162253123002354
  33. Aug 2023
  34. 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
  35. May 2022
  36. www.nature.com www.nature.com
    Saturation variant interpretation using CRISPR prime editing
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    nature.com/articles/s41587-021-01201-1