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Accelerating the Science of SCD Therapies—Is a Cure Possible?

key information

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, USA

DOI: 10.1001/jama.2019.11419

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