Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Accelerating the Science of SCD Therapies—Is a Cure Possible?
source: JAMA
year: 2019
authors: Edward J. Benz Jr, Traci Heath Mondoro, Gary H. Gibbons
summary/abstract:Nearly 7 decades have passed since seminal studies of hemoglobin led to the characterization of sickle cell anemia as the first defined molecular disease. These foundational insights into sickle cell disease (SCD) initiated an exciting era of medicine that has substantially expanded the understanding of the molecular basis of thousands of disorders over the ensuing years. The field of molecular medicine is now at a pivotal moment in its history, a time when scientific capabilities to read, edit, and reprogram the human genetic code for therapeutic approaches are within reach. The rapid pace of innovation in emerging technologies of gene editing and translational research suggest that it is time to accelerate curative therapies for the first defined molecular disease.
organization: Dana-Farber Cancer Institute, USA; National Institutes of Health, USADOI: 10.1001/jama.2019.11419
read more
+myBinderRelated Content
-
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene TherapyA 13-year-old boy with sickle cell disea...
-
Alzheimer’s Treatment Memantine Shows Promise in Treating Sickle Cell DiseaseMemantine, a standard treatment for Alzh...
-
Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical TrialImara’s investigational oral inhibitor...
-
Gene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
Sickle cell disease cure in sights of UAB Stem Cell InstituteTim Townes has a dream. He has a dream t...
-
Stuart H. Orkin, MDDr. Orkin is David G. Nathan Distinguish...
-
A new Sickle Cell Disease Drug Holds Much Promise but Most Sufferers Won’t be Able to Afford itThe regulatory approval of a groundbreak...
Don’t forget to join the oneSCDvoice community!
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.
