While CRISPR gene editing has dominated the genome craze for the last few years, there's a new next-generation platform that's been in the works and is showing big promise for the treatment of a blood disorder.
The system, developed by researchers at Carnegie Mellon University and Yale University, relies on synthetic nucleotide molecules called peptide nucleic acid (PNA). Unlike CRISPR, which uses molecular scissors to ‘cut-and-paste' the desired genetic changes, the PNA system is designed to open up double-stranded DNA. Then, other components of the system coax the host's machinery to repair the faulty genetic change.
In the lab, researchers were able to successfully target a defect in that causes beta thalassemia in a mouse model. The team noted that the system has high specificity for the desired target site and produced a significant change in hemoglobin production.
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