Advances in gene therapy for hemophilia

Hemophilia is a hereditary disorder that can be life-threatening in individuals who have severe spontaneous bleeding resulting from minor trauma or surgery. Although replacement therapy of the missing exogenous factor has improved patients’ quality of life, it has not been possible to establish a lo...

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Bibliographic Details
Published in:Journal of biosciences 2020-12, Vol.45 (1), Article 88
Main Authors: Robles-Rodríguez, Olivia A, Pérez-Trujillo, José J, Villanueva-Olivo, Arnulfo, Villarreal-Martínez, Laura, Marfil-Rivera, Luis J, Rodríguez-Rocha, Humberto, García-García, Aracely, Saucedo-Cárdenas, Odila, Loera-Arias, María J, de Oca-Luna, Roberto Montes
Format: Article
Language:English
Subjects:
Biomedical and Life Sciences
Biomedicine
Bleeding
Cell Biology
Gene therapy
Hemophilia
Life Sciences
Microbiology
Patients
Plant Sciences
Quality of life
Review
Surgery
Sustainable development
Trauma
Zoology
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Summary:Hemophilia is a hereditary disorder that can be life-threatening in individuals who have severe spontaneous bleeding resulting from minor trauma or surgery. Although replacement therapy of the missing exogenous factor has improved patients’ quality of life, it has not been possible to establish a long-term treatment. Due to the severity of the disease and the need for repetitive doses throughout the patient’s life, replacement therapy has become a high-cost treatment option; therefore, the development of self-sustainable long-term therapies is critical. Hemophilia is a good candidate for gene therapy because it is a monogenic disease that can be counteracted by expression of the missing factor. In this article, we review some of the most relevant advances in gene therapy for this illness.
ISSN:0250-5991
0973-7138
DOI:10.1007/s12038-020-00057-y