Thomas Gaj
6PUBLICATIONS
14CO-AUTHORS
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Publications (6)
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|Jan 09, 2026
Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.Katherine Tan, Daniela Del Bosque Siller, Alisha Y Xiong
|Jan 08, 2025
A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD.Tristan X McCallister, Colin K W Lim, Mayuri Singh
|Nov 28, 2024
SPLICER: a highly efficient base editing toolbox that enables in vivo therapeutic exon skipping.Angelo Miskalis, Shraddha Shirguppe, Jackson Winter
|Oct 14, 2023
Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins.M Alejandra Zeballos C, Hayden J Moore, Tyler J Smith
|Jan 19, 2022
Targeted gene silencing in the nervous system with CRISPR-Cas13.Jackson E Powell, Colin K W Lim, Ramya Krishnan
|Dec 28, 2017
In vivo genome editing improves motor function and extends survival in a mouse model of ALS.Thomas Gaj, David S Ojala, Freja K Ekman
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Frequent Collaborators
4 joint publications
Maria A Zeballos
1 joint publications
Freja K Ekman
1 joint publications
Leah C Byrne
1 joint publications
David V Schaffer
1 joint publications
Jackson E Powell
1 joint publications
Tristan X McCallister
1 joint publications
Christian Saporito-Magriña
1 joint publications
Garrett D McPheron
1 joint publications
Ananthan Nambiar
1 joint publications
Sergei Maslov
