5:12 PM
Oct 09, 2018
 |  BC Extra  |  Preclinical News

In utero base editing corrects metabolic birth defect

Researchers from Children's Hospital of Philadelphia and University of Pennsylvania opened the door to prenatal genome editing by using a relatively new CRISPR-based approach called base editor 3 to correct tyrosinemia type I, a rare metabolic birth defect, in utero in mice.

In a paper published Monday in Nature Medicine, the researchers developed a viral vector system for in utero delivery of...

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