40 Matching Annotations
  1. Aug 2026
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    iSCORE-PD: an isogenic stem cell collection to research Parkinson’s disease
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    pmc.ncbi.nlm.nih.gov/articles/PMC13434110/
  3. Jun 2026
  4. www.biorxiv.org www.biorxiv.org
    Identification of Parkinson's disease-associated regulatory variants in human dopaminergic neurons reveals modulators of SCARB2 and BAG3 expression
    1
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    biorxiv.org/content/10.64898/2026.03.26.714241v1
  5. Mar 2026
  6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    ABE9 fused to SpRY Cas9 nickase enables precise generation of bystander free mouse models
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12929607/
  7. Jan 2026
  8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Investigating and correcting a rare pathogenic mutation in GDF11
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12825063/
  9. Nov 2025
  10. www.nature.com www.nature.com
    Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice
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    nature.com/articles/s41551-025-01499-1
  11. Sep 2025
  12. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Custom CRISPR—Cas9 PAM variants via scalable engineering and machine learning
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    pmc.ncbi.nlm.nih.gov/articles/PMC12449813/
  13. Aug 2025
  14. 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/
  15. Jul 2025
  16. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    GenomePAM directs PAM characterization and engineering of CRISPR-Cas nucleases using mammalian genome repeats
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    pmc.ncbi.nlm.nih.gov/articles/PMC12204363/
  17. Jun 2025
  18. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics
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    pmc.ncbi.nlm.nih.gov/articles/PMC11386987/
  19. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Development of a Novel Biomarker Platform for Profiling Key Protein–Protein Interactions to Predict the Efficacy of BH3-Mimetic Drugs
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    pmc.ncbi.nlm.nih.gov/articles/PMC12153628/
  20. May 2025
  21. www.sciencedirect.com www.sciencedirect.com
    Protocol to perform multiplexed assays of variant effect using curated loci prime editing
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    sciencedirect.com/science/article/pii/S2666166725002576
  22. 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
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11997516/
  23. Mar 2025
  24. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    In vivo prime editing rescues photoreceptor degeneration in nonsense mutant retinitis pigmentosa
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11893901/
  25. Dec 2024
  26. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Identification of a novel nuclease activity in human DDX49 helicase
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11651898/
  27. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Engineered transcription-associated Cas9 targeting in eukaryotic cells
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11603292/
  28. www.nature.com www.nature.com
    Glucose limitation protects cancer cells from apoptosis induced by pyrimidine restriction and replication inhibition
    1
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    nature.com/articles/s42255-024-01166-w
  29. www.sciencedirect.com www.sciencedirect.com
    Long-term in vitro expansion of a human fetal pancreas stem cell that generates all three pancreatic cell lineages
    1
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    sciencedirect.com/science/article/pii/S0092867424012546
  30. Nov 2024
  31. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Engineered transcription-associated Cas9 targeting in eukaryotic cells
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC10541143/
  32. Sep 2024
  33. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    ISGylation enhances dsRNA-induced interferon response and NFκB signaling in fallopian tube epithelial cells
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11418117/
  34. www.sciencedirect.com www.sciencedirect.com
    Modeling the atrioventricular conduction axis using human pluripotent stem cell-derived cardiac assembloids
    1
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    sciencedirect.com/science/article/pii/S1934590924002947
  35. www.sciencedirect.com www.sciencedirect.com
    Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells
    1
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    sciencedirect.com/science/article/pii/S1096719224004529
  36. Aug 2024
  37. www.sciencedirect.com www.sciencedirect.com
    The FXR1 network acts as a signaling scaffold for actomyosin remodeling
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    sciencedirect.com/science/article/pii/S009286742400775X
  38. Jul 2024
  39. 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
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11214518/
  40. Jun 2024
  41. 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/
  42. Apr 2024
  43. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Splice modulators target PMS1 to reduce somatic expansion of the Huntington’s disease-associated CAG repeat
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11015039/
  44. Mar 2024
  45. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    In situ architecture of Opa1-dependent mitochondrial cristae remodeling
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10897290/
  46. Feb 2024
  47. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Generation of Human Isogenic Induced Pluripotent Stem Cell Lines with CRISPR Prime Editing
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10880268/
  48. www.biorxiv.org www.biorxiv.org
    iSCORE-PD: an isogenic stem cell collection to research Parkinson’s Disease
    1
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    biorxiv.org/content/10.1101/2024.02.12.579917v1
  49. 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/
  50. Sep 2023
  51. www.sciencedirect.com www.sciencedirect.com
    Base editing corrects the common Salla disease SLC17A5 c.115C>T variant
    1
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    sciencedirect.com/science/article/pii/S2162253123002354
  52. Aug 2023
  53. 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
  54. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    PMS1 as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington’s disease
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10402039/
  55. May 2023
  56. www.sciencedirect.com www.sciencedirect.com
    Generation of three CRISPR/Cas9 edited human induced pluripotent stem cell lines (DHMi005-A-5, DHMi005-A-6 and DHMi005-A-7) carrying a Holt-Oram Syndrome patient-specific TBX5 mutation with known cardiac phenotype and a FLAG-tag after exon 9 of the TBX5 gene
    1
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    sciencedirect.com/science/article/pii/S1873506123001095
  57. Apr 2023
  58. www.biorxiv.org www.biorxiv.org
    Computationally Engineered CRISPR-SpyCas9 High-Fidelity Variants with Improved Specificity and Reduced Non-specific DNA Damage
    1
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    biorxiv.org/content/10.1101/2023.04.11.536265v1
  59. Jan 2023
  60. journals.plos.org journals.plos.org
    In vitro genome editing activity of Cas9 in somatic cells after random and transposon-based genomic Cas9 integration
    1
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    journals.plos.org/plosone/article
  61. Dec 2022
  62. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    A novel intergenic enhancer that regulates Bdnf expression in developing cortical neurons
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC9792897/
  63. Oct 2022
  64. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Highly efficient generation of isogenic pluripotent stem cell models using prime editing
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC9584603/
  65. Jan 2022
  66. www.sciencedirect.com www.sciencedirect.com
    Generation of a CRISPR/Cas edited human induced pluripotent stem cell line DHMi005-A-1 carrying a patient-specific disease-causing point mutation in the TBX5 gene
    2
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    sciencedirect.com/science/article/pii/S187350612200040X
  67. Dec 2020
  68. www.sciencedirect.com www.sciencedirect.com
    Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q
    1
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    sciencedirect.com/science/article/pii/S2211124720314820