[Skip to Navigation]
Scientific Discovery and the Future of Medicine
August 8, 2019

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

Author Affiliations
  • 1Dana-Farber Cancer Institute, Boston, Massachusetts
  • 2National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland
JAMA. 2019;322(10):921-922. doi:10.1001/jama.2019.11419

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.

Limit 200 characters
Limit 25 characters
Conflicts of Interest Disclosure

Identify all potential conflicts of interest that might be relevant to your comment.

Conflicts of interest comprise financial interests, activities, and relationships within the past 3 years including but not limited to employment, affiliation, grants or funding, consultancies, honoraria or payment, speaker's bureaus, stock ownership or options, expert testimony, royalties, donation of medical equipment, or patents planned, pending, or issued.

Err on the side of full disclosure.

If you have no conflicts of interest, check "No potential conflicts of interest" in the box below. The information will be posted with your response.

Not all submitted comments are published. Please see our commenting policy for details.

Limit 140 characters
Limit 3600 characters or approximately 600 words