Corrigen con edición genética un error oculto en el ADN que impide producir una proteína esencial

Researchers at the Severo Ochoa Center for Molecular Biology have successfully corrected a genetic error in human cells that prevents the production of an essential protein. The study focused on addressing a specific, hidden 'misinstruction' within DNA that is responsible for certain cases of propionic acidemia, a rare metabolic disorder.

This experimental breakthrough demonstrates the potential of gene editing technology to rectify underlying genetic defects. By targeting the specific sequence error, the team has provided a proof-of-concept for addressing the molecular roots of this rare disease, marking a significant step in the development of future therapeutic interventions for metabolic conditions.