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

door György, Bence, Lööv, Camilla, Zaborowski, Mikołaj P., Takeda, Shuko, Kleinstiver, Benjamin P., Commins, Caitlin, Kastanenka, Ksenia, Mu, Dakai, Volak, Adrienn, Giedraitis, Vilmantas, Lannfelt, Lars, Maguire, Casey A., Joung, J. Keith, Hyman, Bradley T., Breakefield, Xandra O., Ingelsson, Martin
Gepubliceerd in Mol Ther Nucleic Acids (2018)

Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss

door György, Bence, Nist-Lund, Carl, Pan, Bifeng, Asai, Yukako, Karavitaki, K. Domenica, Kleinstiver, Benjamin, Garcia, Sara P., Zaborowski, Mikołaj P., Solanes, Paola, Spataro, Sofia, Schneider, Bernard, Joung, J. Keith, Geleoc, Gwenaelle S.G., Holt, Jeffrey R., Corey, David P.
Gepubliceerd in Nat Med (2019)

High levels of AAV vector integration into CRISPR-induced DNA breaks

door Hanlon, Killian S., Kleinstiver, Benjamin P., Garcia, Sara P., Zaborowski, Mikołaj P., Volak, Adrienn, Spirig, Stefan E., Muller, Alissa, Sousa, Alexander A., Tsai, Shengdar Q., Bengtsson, Niclas E., Lööv, Camilla, Ingelsson, Martin, Chamberlain, Jeffrey S., Corey, David P., Aryee, Martin J., Joung, J. Keith, Breakefield, Xandra O., Maguire, Casey A., György, Bence
Gepubliceerd in Nat Commun (2019)