9 Matching Annotations
  1. Aug 2026
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Translational reading frame predicts the pathogenicity of C-terminal frameshift deletions in MeCP2
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    pmc.ncbi.nlm.nih.gov/articles/PMC13456685/
  3. Jul 2026
  4. elifesciences.org elifesciences.org
    Translational reading frame determines the pathogenicity of C-terminal frameshift deletions in MeCP2: an alternative therapeutic approach
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    elifesciences.org/reviewed-preprints/109170
  5. Nov 2025
  6. 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
  7. Sep 2025
  8. 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
  9. May 2025
  10. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    DUT (p.Y116C)-Mutation-Induced Thrombocytopenia in Rabbits
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    pmc.ncbi.nlm.nih.gov/articles/PMC12072063/
  11. May 2024
  12. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Context base editing for splice correction of IVSI-110 β-thalassemia
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    ncbi.nlm.nih.gov/pmc/articles/PMC11068610/
  13. Feb 2024
  14. 52.53.155.43 52.53.155.43
    175164.1-20231018101728-covered-e0fd13ba177f913fd3156f593ead4cfd-html.html
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    52.53.155.43/4597aa89381071dcb844ceb7509c9a4dbb75f67d/175164.3-20240103114740-covered-e0fd13ba177f913fd3156f593ead4cfd-html.html
  15. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    CRISPR-Cas9 base editing of pathogenic CaMKIIδ improves cardiac function in a humanized mouse model
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    ncbi.nlm.nih.gov/pmc/articles/PMC10760954/
  16. Dec 2023
  17. 52.53.155.43 52.53.155.43
    175164.1-20231018101728-covered-e0fd13ba177f913fd3156f593ead4cfd-html.html
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    52.53.155.43/4597aa89381071dcb844ceb7509c9a4dbb75f67d/175164.3-20240103114740-covered-e0fd13ba177f913fd3156f593ead4cfd-html.html