"Unlocking the Potential of Junk DNA in Birds for Gene Therapy"

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Source: Phys.org
"Unlocking the Potential of Junk DNA in Birds for Gene Therapy"
Photo: Phys.org
TL;DR Summary

A new gene therapy technique called Precise RNA-mediated INsertion of Transgenes (PRINT) leverages retrotransposons found in birds to safely insert genes into a "safe harbor" in the human genome, avoiding disruption of essential genes or potential cancer risks. This approach complements CRISPR-Cas9 gene editing by providing a method to insert whole genes into the genome, offering promise for treating hereditary diseases caused by various mutations in the same gene. The technique involves using a retroelement protein called R2 to efficiently insert genes into the genome, particularly into the ribosomal RNA encoding region, providing a safe and effective method for gene supplementation.

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