Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model.

Por Shamim H Rahman, Johannes Kuehle, Christian Reimann, Tafadzwa Mlambo, Jamal Alzubi, Morgan L Maeder, Heimo Riedel, Paul Fisch, Tobias Cantz, Cornelia Rudolph, Claudio Mussolino, J Keith Joung, Axel Schambach, Toni Cathomen
Publicado em 2015-05-01

CRISPR-Cas9 treatment partially restores amyloid-β 42/40 in human fibroblasts with the Alzheimer’s disease PSEN1 M146L mutation

Por Evangelos Konstantinidis, Agnieszka Molisak, Florian Perrin, Linn Streubel-Gallasch, Sarah Fayad, Daniel Y. Kim, Karl Petri, Martin J. Aryee, Ximena Aguilar, Bence György, Vilmantas Giedraitis, J. Keith Joung, Vikram Pattanayak, Magnus Essand, Anna Erlandsson, Oksana Berezovska, Martin Ingelsson
Publicado em 2022-06-01

Genome-wide functional perturbation of human microsatellite repeats using engineered zinc finger transcription factors

Por Y. Esther Tak, Gaylor Boulay, Lukuo Lee, Sowmya Iyer, Nicholas T. Perry, Hayley T. Schultz, Sara P. Garcia, Liliane Broye, Joy E. Horng, Shruthi Rengarajan, Beverly Naigles, Angela Volorio, Jeffry D. Sander, Jingyi Gong, Nicolò Riggi, J. Keith Joung, Miguel N. Rivera
Publicado em 2022-04-01