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.

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.

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.

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.