Researchers have for the first time used a gene editing technique to successfully cure a genetic condition in a mouse model. Their findings present a promising new avenue for research into treating genetic conditions during fetal development. 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 a mouse model. Their findings, published in Nature Communications, present a promising new avenue for research into treating genetic conditions during fetal development.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, 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.

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