Hemophilia Gene Therapy: Another Blessing or Another Curse?

Medical therapies for hemophilia patients over the past 60 years have included several blessings and a curse. The long-sought cure with gene therapy may have finally arrived. Unfortunately, preclinical animal models are now raising concerns for genotoxicity with gene therapy. Although no cancers have been detected in humans, it may be a few decades before we know if gene therapy for hemophilia is another blessing, or another curse.
Competing Interests: Disclosure No financial disclosures reported. Artificial intelligence was not used in the study, research, preparation, or writing of this manuscript.
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