Gene Therapy in Hemophilia: Recent Advances

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or IX genes. The palliative treatment of choice based on the use safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable durable concentrations defective circulating factor. Results have so far been encouraging in terms levels times expression using mainly adeno-associated vectors. However, these are associated with immunogenicity hepatotoxicity. Optimizing vector serotypes transgene (variants) boost efficacy, thus increasing viability protocols. It essential that both physicians patients informed about potential benefits risks new therapies, register gene therapy kept information efficacy long-term adverse events treatments administered. In context hemophilia, may result (particularly indirect) cost savings more equitable allocation treatments. case hemophilia A, further research needed into how to effectively package large vector; B, priority should optimize serotype, reducing its hepatotoxicity, transgene, boosting as minimize amount administered decrease incidence without compromising protein expressed.