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
Affiliations
- Jichao Sun
- Department of Pathology and Laboratory Medicine, University of Wisconsin-Madison
- Jared Carlson-Stevermer
- Department of Biomedical Engineering, University of Wisconsin-Madison
- Utpal Das
- Department of Neuroscience, University of California
- Minjie Shen
- Waisman Center, University of Wisconsin-Madison
- Marion Delenclos
- Department of Neuroscience, Mayo Clinic Jacksonville
- Amanda M. Snead
- Taub Institute for Research on Alzheimer’s and the Aging Brain, Columbia University Medical Center
- So Yeon Koo
- Taub Institute for Research on Alzheimer’s and the Aging Brain, Columbia University Medical Center
- Lina Wang
- Department of Pathology and Laboratory Medicine, University of Wisconsin-Madison
- Dianhua Qiao
- Department of Pathology and Laboratory Medicine, University of Wisconsin-Madison
- Jonathan Loi
- Department of Neuroscience, University of Wisconsin-Madison
- Andrew J. Petersen
- Waisman Center, University of Wisconsin-Madison
- Michael Stockton
- Waisman Center, University of Wisconsin-Madison
- Anita Bhattacharyya
- Waisman Center, University of Wisconsin-Madison
- Mathew V. Jones
- Department of Neuroscience, University of Wisconsin-Madison
- Xinyu Zhao
- Waisman Center, University of Wisconsin-Madison
- Pamela J. McLean
- Department of Neuroscience, Mayo Clinic Jacksonville
- Andrew A. Sproul
- Taub Institute for Research on Alzheimer’s and the Aging Brain, Columbia University Medical Center
- Krishanu Saha
- Department of Biomedical Engineering, University of Wisconsin-Madison
- Subhojit Roy
- Department of Pathology and Laboratory Medicine, University of Wisconsin-Madison
- DOI
- https://doi.org/10.1038/s41467-018-07971-8
- Journal volume & issue
-
Vol. 10,
no. 1
pp. 1 – 11
Abstract
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.