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
Related Content
-
A CRISPR Focus on Attitudes and Beliefs Toward Somatic Genome Editing From Stakeholders Within the Sickle Cell Disea...Purpose: Genome editing holds both trem...
-
Acceptability of In Utero Hematopoietic Cell Transplantation for Sickle Cell DiseaseBACKGROUND: In utero hematopoietic cell ...
-
Calimmune Expands Lentiviral Gene Therapy Pipeline Through License of Sickle Cell Disease Therapeutic CandidateLicense of Gamma-Globin from Cincinnati ...
-
Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Ga...Acetylon Pharmaceuticals, Inc., the lead...
-
MaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
-
NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and TrialsWide interest in a CBS 60 Minutes story ...
-
Solution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
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.