There are many human genetic diseases — for example, sickle-cell disease, cystic fibrosis and Hutchinson-Gilford progeria syndrome — that currently have no cure and are caused by point mutations, for which there is a pressing need to develop precision genome editing tools able to correct these mutations with high efficiency and accuracy.
Assistant Professor Audrey Lapinaite from Arizona State University’s School of Molecular Sciences and the Biodesign Institute’s ASU-Banner Neurodegenerative Disease Research Center is working on this problem and has just been awarded a prestigious National Institutes of Health (NIH) Director’s New Innovator Award.Continue reading