Title: 13th C1-inhibitor deficiency and angioedema workshop—2023
Abstract: The vision of modifying the human genome to cure disease has been pursued since the 1980s.It is only recently however, that the fruits of these efforts started to reach clinical practice and transform patient lives.Among the few gene therapies that have obtained FDA approval are HEMGENIX for the treatment of adults with congenital Factor IX deficiency (Hemophilia B), LUXTURNA for the treatment of adults with retinal dystrophy caused by biallelic RPE65 mutations, ZYNGEGLO for the treatment of adults with beta-thalasemia, and ZOLGENSMA for treatment of children less than 2 years with spinal muscular atrophy caused by biallelic SMN1 mutations.These gene therapy approaches