Clinical trials of genome editing to treat sickle cell anemia must be designed with input from patients and their parents and physicians, a recent study by National Human Genome Research Institute (NHGRI) scientists concluded.
Two methods of CRISPR (clustered regularly interspaced short palindromic repeats) somatic genome editing that could substantially reduce the symptoms of or potentially cure sickle cell disease—which affects more than 100 000 US residents—are being investigated, the authors wrote. Clinical trials will be necessary to advance the potential treatment, but, if not adequately informed, seriously ill patients could overestimate the benefits of early-phase studies while underestimating the risks.
Rubin R. Importance of Patient Input in Designing CRISPR Trials. JAMA. 2020;324(20):2019. doi:10.1001/jama.2020.22597
Coronavirus Resource Center
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: