Article

Hemophilia gene therapy comes of age

2017-12-08

Abstract excerpt

Abstract Concurrent with the development of recombinant factor replacement products, the characterization of the F9 and F8 genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first “cure...

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Literature Corpus work: c0993554-08da-5729-a2b0-40eff9c2a0f8
DOI: 10.1182/bloodadvances.2017009878

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