Gene therapy for hemophilia: Current status and future perspectives
Department of Genetics FAMEN UJED, Av. Universidad sin número zona centro CP 34000, Durango, Dgo México.
Review Article
GSC Advanced Research and Reviews, 2023, 15(03), 295–303.
Article DOI: 10.30574/gscarr.2023.15.3.0192
Publication history:
Received on 30 April 2023; revised on 23 June 2023; accepted on 26 June 2023
Abstract:
Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clotting. Gene therapy for hemophilia involves the introduction of a healthy gene into the patient's cells to produce the missing protein.
There are two main types of gene therapy for hemophilia: ex vivo gene therapy, which involves the extraction of cells from the patient, introduction of the healthy gene into these cells in the laboratory, and reintroduction of the modified cells into the patient; and in vivo gene therapy, which involves the direct delivery of the healthy gene into the patient's body using viral vectors.
However, there are still challenges in the large-scale implementation of gene therapy, including safety and long-term efficacy, and the costs associated with the technology.
Keywords:
Gene therapy; Hemophilia; Bleeding disorder; Hemophilia A and B
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Copyright © 2023 Author(s) retain the copyright of this article. This article is published under the terms of the Creative Commons Attribution Liscense 4.0
