Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Fetal Haemoglobin in Sickle-cell Disease: From Genetic Epidemiology to New Therapeutic Strategies
source: Lancet
year: 2016
authors: Lettre G, Bauer DE
summary/abstract:Sickle-cell disease affects millions of individuals worldwide, but the global incidence is concentrated in Africa. The burden of sickle-cell disease is expected to continue to rise over the coming decades, adding to stress on the health infrastructures of many countries. Although the molecular cause of sickle-cell disease has been known for more than half a century, treatment options remain greatly limited. Allogeneic haemopoietic stem-cell transplantation is the only existing cure but is limited to specialised clinical centres and remains inaccessible for most patients. Induction of fetal haemoglobin production is a promising strategy for the treatment of sickle-cell disease. In this Series paper, we review scientific breakthroughs in epidemiology, genetics, and molecular biology that have brought reactivation of fetal haemoglobin to the forefront of sickle-cell disease research. Improved knowledge of the regulation of fetal haemoglobin production in human beings and the development of genome editing technology now support the design of innovative therapies for sickle-cell disease that are based on fetal haemoglobin.
organization: Montreal Heart Institute, Montreal, QC, Canada; Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School and Harvard Stem Cell Institute, Boston, MA, USADOI: 10.1016/S0140-6736(15)01341-0
read more full text
+myBinderRelated Content
-
Sickle Cell Disease Research Shows Progress in Preventing Related Complications and DeathStudies aim to limit pain crises, preven...
-
Kids Who Need Sickle Cell Meds Don’t Always Get ThemLess than a fifth of U.S. children with ...
-
What it’s like to be 17 and living with sickle cell diseaseTre Adegoroye is 17 and was diagnosed wi...
-
MARAC Statement: Crizanlizumab (Adakveo)July 7, 2023 - SCDAA’s Medical and Res...
-
Spectra Optia Device Save Way to Treat Sickle Cell While Using Less Blood Cell Packs, Study FindsThe blood-separation device Spectra Opti...
-
GBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of Sickle Cell Disease (SCD)Global Blood Therapeutics, Inc. (GBT) to...
-
Preoperative blood transfusions for sickle cell diseaseBackground: Sickle cell disease is one ...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.