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
Related Content
-
Immunogenicity and Safety of 10-valent Pneumococcal Nontypeable Haemophilus influenzae Protein D Conjugate Vaccine (...BACKGROUND: Immunogenicity, safety and r...
-
First World Cord Blood Day on Nov. 15 to Highlight Cord Blood Uses, Stem Cell ResearchThe inaugural World Cord Blood Day on No...
-
Sam Rodgers-Melnick, MT-BCSamuel Rodgers-Melnick, MT-BC is a music...
-
CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
-
A new Sickle Cell Disease Drug Holds Much Promise but Most Sufferers Won’t be Able to Afford itThe regulatory approval of a groundbreak...
-
A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive EventsBACKGROUND: Sickle cell anemia is an inh...
-
Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
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.