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In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia

28 Mar 2024

Abstract excerpt

Hemophilia is a genetic disorder that is caused by mutations in coagulation factor VIII (hemophilia A) or IX (hemophilia B) genes resulting in blood clotting disorders. Despite advances in therapies, such as recombinant proteins and products with extended half-lives, the treatment of hemophilia still faces two major limitations: the short duration of therapeutic effect and production of neutralizing antibodies...

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PMID: 38538969
DOI: 10.1007/s40291-024-00705-1

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