CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington’s Disease Mouse Model von Ekman, Freja K., Ojala, David S., Adil, Maroof M., Lopez, Paola A., Schaffer, David V., Gaj, Thomas

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hPSC-Derived Striatal Cells Generated Using a Scalable 3D Hydrogel Promote Recovery in a Huntington Disease Mouse Model von Adil, Maroof M., Gaj, Thomas, Rao, Antara T., Kulkarni, Rishikesh U., Fuentes, Christina M., Ramadoss, Gokul N., Ekman, Freja K., Miller, Evan W., Schaffer, David V.

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High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition von Selvaraj, Sridhar, Feist, William N., Viel, Sebastien, Vaidyanathan, Sriram, Dudek, Amanda M., Gastou, Marc, Rockwood, Sarah J., Ekman, Freja K., Oseghale, Aluya R., Xu, Liwen, Pavel-Dinu, Mara, Luna, Sofia E., Cromer, M. Kyle, Sayana, Ruhi, Gomez-Ospina, Natalia, Porteus, Matthew H.

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Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery von Gaj, Thomas, Staahl, Brett T, Rodrigues, Gonçalo M C, Limsirichai, Prajit, Ekman, Freja K, Doudna, Jennifer A, Schaffer, David V

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In vivo genome editing improves motor function and extends survival in a mouse model of ALS von Gaj, Thomas, Ojala, David S, Ekman, Freja K, Byrne, Leah C, Limsirichai, Prajit, Schaffer, David V

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