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Targeted Gene Editing Cures Blood Disorder in Fetal Mice

Provided by UConn Communications

Nanoparticle circulation in an extraembryonic vein (larger vessel) and artery (smaller vessel) three hours after injection. (Gif supplied by Ramon Bahal) Nanoparticle circulation in an extraembryonic vein (larger vessel) and artery (smaller vessel) three hours after injection. Photo credit:(Media/Yale University)

A team of researchers, including UConn assistant professor of pharmaceutics Raman Bahal, has, for the first time, corrected a genetic mutation in a mammalian fetus using a targeted gene editing technique. The approach offers a potential new pathway for treating inherited genetic disorders during the earliest stages of development.

Every year, an estimated 8 million children are born with severe genetic disorders or birth defects. While genetic conditions can be detected during pregnancy using amniocentesis, there are no treatment options currently to correct the conditions before birth. Read more.

Published July 26th, 2018 by UConn Today


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