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Items: 3

1.

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

Adil MM, Gaj T, Rao AT, Kulkarni RU, Fuentes CM, Ramadoss GN, Ekman FK, Miller EW, Schaffer DV.

Stem Cell Reports. 2018 May 8;10(5):1481-1491. doi: 10.1016/j.stemcr.2018.03.007. Epub 2018 Apr 5.

2.

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

Gaj T, Ojala DS, Ekman FK, Byrne LC, Limsirichai P, Schaffer DV.

Sci Adv. 2017 Dec 20;3(12):eaar3952. doi: 10.1126/sciadv.aar3952. eCollection 2017 Dec.

3.

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

Gaj T, Staahl BT, Rodrigues GMC, Limsirichai P, Ekman FK, Doudna JA, Schaffer DV.

Nucleic Acids Res. 2017 Jun 20;45(11):e98. doi: 10.1093/nar/gkx154.

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