Scientists from Beam Therapeutics (Cambridge, USA) have for the first time successfully corrected a mutation in a human gene with a single injection. The achievement was made possible by a DNA base editing technology, in which nine patients had an incorrect part of their genetic sequence replaced.
As a result of the intervention, the functions of the damaged gene were restored, which may slow down or stop the progression of a rare disease affecting the liver and lungs. The key novelty is the effectiveness after one administration, distinguishing this approach from previous methods that required multiple injections.
This breakthrough could become the basis for a new era of therapy for inherited diseases, offering the possibility of precise and safer intervention in a person’s DNA.
