Nanoparticles Delivery Synthetic DNA in Treating Thalassemia in Lab Animals
Several years ago a gene repair technique called “chimeraplasty” looked promising, though it never reached fruition. The idea was to create an oligo that would target a specific location, but have a single base mismatch which the researchers hoped would invoke a DNA repair mechanism. But, now, Yale scientists have used a similar approach and corrected thalassemia in lab mice. According to this piece: “The team identified a protein in bone marrow that can activate stem cells. They combined the protein with synthetic molecules that act like DNA and encased the combo in nanoparticles that they delivered to mice via intravenous injection,. . . .. The synthetic DNA, known as PNAs, bring about gene editing by binding to the target gene and prompting the cell to repair the mutation.” If this works in humans, it may solve one of the perplexing problems of CRISPR gene editing—that of “off targeting.” MORE
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