Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 1992 Dec;157(6):631-6.

Future prospects for treatment of hemoglobinopathies

J A Stamatoyannopoulos  1
Affiliations
Review

Future prospects for treatment of hemoglobinopathies

J A Stamatoyannopoulos. West J Med. 1992 Dec.

Abstract

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.

PubMed Disclaimer

Comment in

References

    1. Blood. 1992 May 15;79(10):2555-65 - PubMed
    1. Ann N Y Acad Sci. 1985;445:188-97 - PubMed
    1. EMBO J. 1990 Jan;9(1):233-40 - PubMed
    1. Proc Natl Acad Sci U S A. 1988 Nov;85(22):8540-2 - PubMed
    1. Blood. 1988 Dec;72(6):1961-7 - PubMed