The Unexpected Key to Safe Gene Therapy: Bird Junk DNA

Retrotransposons can insert new genes into a “safe harbor” in the genome, complementing CRISPR gene editing.

The recent greenlighting of a CRISPR-Cas9 treatment for sickle cell disease underscores the efficacy of gene editing technologies in deactivating genes to heal inherited illnesses. However, the capability to integrate entire genes into the human genome as replacements for faulty or harmful ones remains unachievable.

A new technique that employs a retrotransposon from birds to insert genes into the genome holds more promise for gene therapy, since it inserts genes into a “safe harbor” in the human genome where the insertion won’t disrupt essential genes or lead to cancer.