“Junk DNA” in Birds May Hold Key to Safe, Efficient Gene Therapy

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

Retrotransposons found in the genomes of the white-throated sparrow and the zebra finch are shown to safely shepherd transgenes into the human genome, providing a gene therapy approach complementary to CRISPR-Cas9 gene editing. (Credit: Briana Van Treeck, UC Berkeley).

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease. But it's still not possible to insert whole genes into the human genome to substitute for defective or deleterious genes. A new technique that employs a retrotransposon from birds to insert genes into the genome holds more promise for gene therapy, because it inserts genes into a "safe harbor" in the human genome where the insertion won't disrupt essential genes or lead to cancer.

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