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

por Gaj, Thomas, Ojala, David S., Ekman, Freja K., Byrne, Leah C., Limsirichai, Prajit, Schaffer, David V.
Publicado 2017

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

por Ekman, Freja K., Ojala, David S., Adil, Maroof M., Lopez, Paola A., Schaffer, David V., Gaj, Thomas
Publicado 2019

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

por Gaj, Thomas, Staahl, Brett T., Rodrigues, Gonçalo M. C., Limsirichai, Prajit, Ekman, Freja K., Doudna, Jennifer A., Schaffer, David V.
Publicado 2017

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

por 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.
Publicado 2018