January 3, 2019
In the first study of its kind, researchers explored patients', parents' and physicians' perspectives on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease (SCD). CRISPR-Cas9 is a gene editing technology that researchers have harnessed to explore two approaches for reducing the burden of the disease. The first approach will let researchers correct the SCD mutation by removing and replacing it with a normal gene, while the second approach increases the production of fetal hemoglobin which reduces disease severity. Study participants expressed overall optimism about participating in human genome editing clinical trials, but were concerned about treatment risks and transparency of the research enterprise, according to National Institutes of Health researchers and their colleagues. The findings were published December 24 in Genetics in Medicine.
Sickle cell anemia