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
-
Determining Adherence to Quality Indicators in Sickle Cell Anemia Using Multiple Data SourcesINTRODUCTION: Advances in primary prophy...
-
Patterns of opioid use in sickle cell diseasePain, the hallmark complication of sickl...
-
Jazz’ Journey With Sickle Cellhttps://www.youtube.com/watch?v=OMC5wOyD...
-
GBT announces positive top-line data from part A of the phase 3 HOPE study of Voxelotor in sickle cell diseaseGlobal Blood Therapeutics, Inc. today an...
-
NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and TrialsWide interest in a CBS 60 Minutes story ...
-
Sickle cell patient with severe anemia rapidly improves with voxelotor, case study showsGlobal Blood Therapeutics (GBT) reported...
-
APS Scientific Meeting 2019The American Pain Society will hold its ...
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.