CRISPR/Cas9 editing of APP C-terminus attenuates β-cleavage and promotes α-cleavage

oleh: Jichao Sun, Jared Carlson-Stevermer, Utpal Das, Minjie Shen, Marion Delenclos, Amanda M. Snead, So Yeon Koo, Lina Wang, Dianhua Qiao, Jonathan Loi, Andrew J. Petersen, Michael Stockton, Anita Bhattacharyya, Mathew V. Jones, Xinyu Zhao, Pamela J. McLean, Andrew A. Sproul, Krishanu Saha, Subhojit Roy

Format: Article
Diterbitkan: Nature Portfolio 2019-01-01

Deskripsi

Gene editing strategies are typically designed to correct mutant genes, but most neurodegenerative diseases are sporadic. Here the authors describe a strategy to selectively edit the C-terminus of APP and attenuate amyloid-β production, while upregulating neuroprotective α-cleavage.