Advances in gene therapy for hemophilia
OLIVIA A ROBLES-RODRI´GUEZ JOSE´ J PE´REZ-TRUJILLO ARNULFO VILLANUEVA-OLIVO LAURA VILLARREAL-MARTI´NEZ LUIS J MARFIL-RIVERA HUMBERTO RODRI´GUEZ-ROCHA ARACELY GARCI´A-GARCI´A ODILA SAUCEDO-CA´ RDENAS MARI´A J LOERA-ARIAS ROBERTO MONTES DE OCA-LUNA
Hemophilia is a hereditary disorder that can be life-threatening in individuals who have severe spontaneousbleeding resulting from minor trauma or surgery. Although replacement therapy of the missing exogenousfactor has improved patients’ quality of life, it has not been possible to establish a long-term treatment. Due tothe severity of the disease and the need for repetitive doses throughout the patient’s life, replacement therapyhas become a high-cost treatment option; therefore, the development of self-sustainable long-term therapies iscritical. Hemophilia is a good candidate for gene therapy because it is a monogenic disease that can becounteracted by expression of the missing factor. In this article, we review some of the most relevant advancesin gene therapy for this illness.
OLIVIA A ROBLES-RODRI´GUEZ1 JOSE´ J PE´REZ-TRUJILLO1 ARNULFO VILLANUEVA-OLIVO1 LAURA VILLARREAL-MARTI´NEZ2 LUIS J MARFIL-RIVERA2 HUMBERTO RODRI´GUEZ-ROCHA1 ARACELY GARCI´A-GARCI´A1 ODILA SAUCEDO-CA´ RDENAS1 3 MARI´A J LOERA-ARIAS1 ROBERTO MONTES DE OCA-LUNA1
Volume 45, 2020
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