Nature Communications (Jan 2019)

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

  • 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

DOI
https://doi.org/10.1038/s41467-018-07971-8
Journal volume & issue
Vol. 10, no. 1
pp. 1 – 11

Abstract

Read online

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.