CHARACTERIZATION AND FUNCTIONAL ANALYSIS OF THE CALMODULIN-BINDING DOMAIN OF RAC1 GTPASE.




In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice

Gene therapy and rebalancing therapy have emerged as promising approaches for treating hemophilia A, but there are limitations, such as temporary efficacy due to individual differences.Genome editing for hemophilia has shown long-term therapeutic potential in preclinical trials.However, a cautious approach is necessary because genome editing is irr

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