Single Nucleotide Edit Fixes Heart Muscle Disease

Genetically editing a single nucleotide base could clinically correct cardiomyopathies, preclinical research suggests.

The study, in the Proceedings of the National Academy of Sciences , demonstrates the power of base editors to correct disease-causing point mutations, where a solitary change in the DNA sequence can have devastating consequences.

Using the well-established CRISPR/Cas system, the base editing process enables single nucleotide changes without introducing double-strand DNA breaks.

In the current research, it was able to correct cardiac disease caused by mutations in Lamin A ( LMNA ) gene, which codes for membrane proteins separating the nucleus and the rest of a cell.

“Our work represents an important step toward the potential clinical correction of cardiomyopathies