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Thomas Gaj

Scripps Research Institute
University of Illinois at Urbana–Champaign
University of California, Berkeley

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Next-Generation CRISPR Technologies and Their Applications in Gene and Cell Therapy.

2020 Trends in Biotechnology
- Thomas Gaj
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Treatment of a mouse model of ALS by in vivo base editing

2020 Molecular Therapy
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CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington’s disease mouse model

2019 Molecular therapy. Nucleic acids
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The continuously evolving CRISPR barcoding toolbox

2018 Genome Biology
- Thomas Gaj
- Pablo Perez-Pinera
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Manufacturing and Delivering Genome-Editing Proteins

2018 Methods of Molecular Biology
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hPSC-Derived Striatal Cells Generated Using a Scalable 3D Hydrogel Promote Recovery in a Huntington Disease Mouse Model

2018 Stem cell reports
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A Hypothalamic Switch for REM and Non-REM Sleep

2018 Neuron
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Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery

2017 Nucleic Acids Research
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In vivo genome editing improves motor function and extends survival in a mouse model of ALS

2017 Science Advances
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125. Purging Latent HIV Infection by CRISPR-Mediated Viral Reactivation

2016 Molecular Therapy
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