Advances in gene therapy for hemophilia.
J Biosci
; 452020.
Article
em En
| MEDLINE
| ID: mdl-32661215
ABSTRACT
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.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Hemofilia A
/
Hemorragia
Limite:
Humans
Idioma:
En
Revista:
J Biosci
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
México