Future prospects for treatment of hemoglobinopathies.
West J Med
; 157(6): 631-6, 1992 Dec.
Article
en En
| MEDLINE
| ID: mdl-1282285
Strategies for the treatment of sickle cell anemia and beta-thalassemia are founded on the knowledge that these disorders result from structural or functional defects in an adult gene for which an intact fetal counterpart exists. During the past decade, several pharmacologic agents have been investigated for their potential to ameliorate sickle cell anemia and beta-thalassemia by increasing the synthesis of fetal hemoglobin in adults. Progress in understanding globin gene regulation is now being combined with advances in retrovirus-mediated gene transfer, and the once-distant goal of providing gene therapy for hemoglobinopathies is rapidly approaching reality.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Talasemia beta
/
Anemia de Células Falciformes
Límite:
Adult
/
Humans
Idioma:
En
Revista:
West J Med
Año:
1992
Tipo del documento:
Article
Pais de publicación:
Estados Unidos