Many approaches to correct these mutations in patients’ own cells are being tested, but they often rely on making breaks in the gene that can lead to unwanted DNA changes. Prime editing employs a “search and replace” strategy to convert the disease-causing mutation in the gene back to a normal sequence while avoiding the DNA breaks that can be problematic.
In blood stem cells from SCD patients, prime editing was able to efficiently and specifically correct the genetic deficit in the gene, with a conversion of 15% to 41%. Transplanted into mice, the edited cells led to the production of healthy hemoglobin and less red blood cell sickling in low oxygen conditions.
The researchers believe that prime editing and transplanting patient stem cells may represent a promising therapeutic strategy for SCD, as well as other genetic diseases. However, more studies are needed to ensure its safety, and the approach’s time-consuming nature and need for optimization could limit its potential.
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