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https://www.ias.ac.in/article/fulltext/jbsc/045/0088

Coagulation; gene editing; gene therapy; hemophilia

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´ RDENAS^{1 3} MARI´A J LOERA-ARIAS¹ ROBERTO MONTES DE OCA-LUNA¹

1. Departamento de Histologı´a, Facultad de Medicina, Universidad Auto´noma de Nuevo Leo´n, C.P. 64460 Monterrey, Mexico
2. Servicio de Hematologı´a, Hospital Universitario ‘‘Dr. Jose´ Eleuterio Gonza´ lez Gonza´ lez’’, Universidad Auto´noma de Nuevo Leo´n, C.P. 64460 Monterrey, Mexico
3. Departamento de Gene´tica Molecular, Centro de Investigacio´n Biome´dica del Noreste (CIBIN), Instituto Mexicano del Seguro Social (IMSS), C.P. 64720 Monterrey, Mexico

Published

2 July 2020