Author

T. Gaj

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Bioengineering· 4

Signature Assays, Organisms & Methods

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Organism:mouse· 4 Research Focus:drug response / drug discovery· 3 Approach:genetic perturbation / CRISPR· 3 Assay:CRISPR screen· 2 Research Focus:cell signaling & trafficking· 1 Research Focus:chromatin & epigenetic regulation· 1 Approach:deep learning· 1 Approach:experimental assay· 1

Frequent Co-Authors

J. E. Powell· 3 shared C. K. W. Lim· 2 shared P. Perez-Pinera· 2 shared S. Zhang· 2 shared T. X. McCallister· 2 shared A. Miskalis· 1 shared A. Nambiar· 1 shared A. Zeballos· 1 shared

Activity by Year

2022-2024 · peak 2/yr

Co-Author Collaboration Network

10 top collaborators · 23 mutual co-author links

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3 shared papersBioengineering

J. E. Powell

Mutual co-authors in cluster

Author profile →

4 papers

Recent·Most cited

SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping

A. Miskalis, S. Shirguppe, J. Winter, G. Elias, D. Swami, A. Nambiar, M. Stilger, W. Woods, N. Gosstola, M. Gapinske, A. Zeballos, H. Moore, S. Maslov, T. Gaj, P. Perez-Pinera

SPLICER improves exon skipping by editing both splice acceptor and donor sites, reducing aberrant splicing and enabling therapeutic applications in Alzheimer's disease.

bioRxiv · Apr 1, 2024 · PreprintBioengineering

A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD

T. X. McCallister, C. K. W. Lim, W. M. Terpstra, M. A. Z. C., S. Zhang, J. E. Powell, T. Gaj

This study develops a high-fidelity CRISPR-Cas13 system that reduces C9ORF72 repeat RNA expression in a rodent model, decreasing RNA foci and reversing transcriptional deficits without affecting normal C9ORF72 levels.

bioRxiv · Dec 12, 2023Bioengineering

Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins

M. A. Zeballos C., H. J. Moore, T. J. Smith, J. E. Powell, N. S. Ahsan, S. Zhang, T. Gaj

This study uses RNA-targeting CRISPR effector proteins to reduce TDP-43 pathology by targeting ataxin-2 in a mouse model, improving function and survival.

bioRxiv · Apr 7, 2023Bioengineering

CRISPR base editing of cis-regulatory elements enables target gene perturbations

C. K. W. Lim, T. X. McCallister, C. Saporito-Magrina, G. D. McPheron, R. Krishnan, M. A. Zeballos, J. E. Powell, L. V. Clark, P. Perez-Pinera, T. Gaj

This study shows CRISPR base editors can reduce gene expression by editing cis-regulatory elements, with examples in human and mouse models.

bioRxiv · May 26, 2022 · PreprintBioengineering