PNA-Based Gene Editing Technique Cures Genetic Disorder in Utero in Mouse Model; Technique Show No Off-Target Effects Suggesting Advantage Over CRISPR/Cas9 for Clinical Uses

Researchers at Carnegie Mellon University and Yale University have, for the first time, used a gene editing technique to successfully cure a genetic condition in utero in a mouse model. Their findings, published online on June 26, 2018 in Nature Communications, present a promising new avenue for research into treating genetic conditions during fetal development. The open-access article is titled “In Utero Nanoparticle Delivery for Site-Specific Genome Editing.” An estimated 8 million children are born each year with severe genetic disorders or birth defects. Genetic conditions can often be detected during pregnancy using amniocentesis, but there are no treatment options to correct these genetic conditions before birth. “Early in embryonic development, there are a lot of stem cells dividing at a rapid pace. If we can go in and correct a genetic mutation early on, we could dramatically reduce the impact the mutation has on fetal development or even cure the condition,” said Danith Ly, PhD, Professor of Chemistry in Carnegie Mellon’s Mellon College of Science. In this study, the researchers used a peptide nucleic acid-based gene editing technique ( that they had previously used to cure beta thalassemia, a genetic blood disorder that results in the reduced production of hemoglobin, in adult mice, using intravenous administration of the PNAs. Peptide nucleic acids (PNAs) are synthetic molecules that combine a synthetic protein backbone with the nucleobases found in DNA and RNA. The PNAs used in this study were created by Dr. Ly at Carnegie Mellon’s Center for Nucleic Acids Science and Technology (CNAST), a leading center for PNA science. Their technique uses an FDA-approved nanoparticle to deliver PNA molecules paired with donor DNA to the site of a genetic mutation.
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