The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease. However, researchers have now shown that there is a risk of inadvertently ...
This is because they can be used to correct specific defective sections of the genome. Unfortunately, however, there is a catch: under certain conditions, the repair can lead to new genetic defects – ...
Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo treatments capable of precisely and durably engineering the hematopoietic system, today unveiled its ...
More information: Vera Bzhilyanskaya et al, High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease, Science Translational Medicine (2024).