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Gene therapy in haemophilia: literature review and regional perspectives for Turkey.
Kavakli, Kaan; Antmen, Bülent; Okan, Vahap; Sahin, Fahri; Aytaç, Selin; Balkan, Can; Berber, Ergül; Kaya, Zühre; Küpesiz, Alphan; Zülfikar, Bülent.
Affiliation
  • Kavakli K; Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Bornova, 35100 Izmir, Turkey.
  • Antmen B; Division of Hematology, Department of Pediatrics, Acibadem Adana Hospital, Adana, Turkey.
  • Okan V; Division of Hematology, Department of Internal Diseases, Gaziantep University Faculty of Medicine, Gaziantep, Turkey.
  • Sahin F; Division of Hematology, Department of Internal Diseases, Ege Adult Hemophilia and Thrombosis Center, Ege University Faculty of Medicine, Izmir, Turkey.
  • Aytaç S; Division of Hematology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey.
  • Balkan C; Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey.
  • Berber E; Department of Molecular Biology and Genetics, Istanbul Arel University, Istanbul, Turkey.
  • Kaya Z; Division of Hematology, Department of Pediatrics, Gazi University Faculty of Medicine, Ankara, Turkey.
  • Küpesiz A; Division of Hematology, Department of Pediatrics, Akdeniz University Faculty of Medicine, Antalya, Turkey.
  • Zülfikar B; Division of Hematology, Department of Pediatrics, Istanbul University Faculty of Medicine, Istanbul, Turkey.
Ther Adv Hematol ; 13: 20406207221104591, 2022.
Article in En | MEDLINE | ID: mdl-35898436
Haemophilia is an X-linked lifelong congenital bleeding disorder that is caused by insufficient levels of factor VIII (FVIII; haemophilia A) or factor IX (FIX; haemophilia B) and characterized by spontaneous and trauma-related bleeding episodes. The cornerstone of the treatment, factor replacement, constitutes several difficulties, including frequent injections due to the short half-life of recombinant factors, intravenous administration and the risk of inhibitor development. While extended half-life factors and subcutaneous novel molecules enhanced the quality of life, initial successes with gene therapy offer a significant hope for cure. Although adeno-associated viral (AAV)-based gene therapy is one of the most emerging approaches for treatment of haemophilia, there are still challenges in vector immunogenicity, potency and efficacy, genotoxicity and persistence. As the approval for the first gene therapy product is coming closer, eligibility criteria for patient selection, multidisciplinary approach for optimal delivery and follow-up and development of new pricing policies and reimbursement models should be concerned. Therefore, this review addresses the unmet needs of current haemophilia treatment and explains the rationale and principles of gene therapy. Limitations and challenges are discussed from a global and national perspective and recommendations are provided to adopt the gene therapies faster and more sufficient for the haemophilia patients in developing countries like Turkey.
Key words

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Guideline / Prognostic_studies / Systematic_reviews Language: En Journal: Ther Adv Hematol Year: 2022 Type: Article Affiliation country: Turkey

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Guideline / Prognostic_studies / Systematic_reviews Language: En Journal: Ther Adv Hematol Year: 2022 Type: Article Affiliation country: Turkey