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 no Sci Adv (2017)

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 no Nucleic Acids Res (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 no Stem Cell Reports (2018)