Terapia Génica como tratamiento para la hemofilia

Abstract

Introduction: Hemophilia is a hereditary hemorrhagic disease caused by a deficiency of coagulation factors VIII (Hemophilia A) and IX (Hemophilia B). It is characterized by the presence of spontaneous systemic bleeding, but predominantly in joints and soft tissues. It is currently a manageable condition. Our goal is to control and prevent bleeding. This has been possible through various treatments such deficient factor infusion, long half-life factors, therapy not based on coagulation factors and lately the gene therapy that allows for long-term factor expression.

General Objective: Collect information about the efficacy of gene therapy as a treatment for hemophilia.

Methodology: This research is a descriptive bibliographic review, which includes articles whose publication date is at least five years old, by searching scientific databases, such as PubMed/Medline, ScienceDirect, Scopus, etc. And use of Boolean operators to acquire more information.

Results: In both type A and B hemophilia, gene therapy is involved in significantly increasing the expression of factor VIII and IX, which causes a reduction in the amount of bleeding and prophylactic use of factor.