Freja K. Ekman

Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery

Jennifer A. Doudna, David V. Schaffer, Thomas Gaj, 2017, Nucleic acids research.

CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington’s Disease Mouse Model

David V. Schaffer, Thomas Gaj, D. Schaffer, 2019, Molecular therapy. Nucleic acids.

High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition.

M. Porteus, M. Gastou, W. Feist, 2023, Nature biotechnology.

hPSC-Derived Striatal Cells Generated Using a Scalable 3D Hydrogel Promote Recovery in a Huntington Disease Mouse Model

Evan W. Miller, D. Schaffer, Maroof M. Adil, 2018, Stem cell reports.

In vivo genome editing improves motor function and extends survival in a mouse model of ALS

David V Schaffer, Thomas Gaj, D. Schaffer, 2017, Science Advances.