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Gene Therapy in Hemophilia: Recent Advances

17 Jul 2021

Abstract excerpt

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable and durable concentrations of the defective circulating factor. Results have so far been encouraging in terms of levels and times of expression using mainly...

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PMID: 34299267
DOI: 10.3390/ijms22147647

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