Korrektur von Hämophilie-B-Mutationen mit CRISPR/Cas9: Präklinische Erkenntnisse
CRISPR/Cas9 gene editing holds promise for treating genetic disorders like hemophilia B. Preclinical studies demonstrate the feasibility of correcting disease-causing mutations and restoring clotting factor IX expression. These findings pave the way for potential clinical applications to alleviate the burden of hemophilia B.





