CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer’s Disease

Authors: Bence György, Camilla Lööv, Mikołaj Piotr Zaborowski, Shuko Takeda, Benjamin P. Kleinstiver, Caitlin Commins, Ksenia V. Kastanenka, Dakai Mu, Adrienn Volak, Vilmantas Giedraitis, Lars Lannfelt, Casey A. Maguire, J. Keith Joung, Bradley T. Hyman, Xandra O. Breakefield, Martin Ingelsson - Molecular Therapy — Nucleic Acids 2018 cited by 189

allele both ex vivo and in vivo-and thereby decrease pathogenic Aβ. Hence, this system may have the potential to be developed as a tool for gene therapy against AD caused by APPswe and other point mutations associated with increased Aβ.