Gaj T - Search Results

1

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

McCallister TX, Lim CKW, Singh M, Zhang S, Ahsan NS, Terpstra WM, Xiong AY, Zeballos C MA, Powell JE, Drnevich J, Kang Y, Gaj T. McCallister TX, et al. Among authors: gaj t. Nat Commun. 2025 Jan 8;16(1):460. doi: 10.1038/s41467-024-55548-5. Nat Commun. 2025. PMID: 39779681 Free PMC article.

2

SPLICER: a highly efficient base editing toolbox that enables in vivo therapeutic exon skipping.

Miskalis A, Shirguppe S, Winter J, Elias G, Swami D, Nambiar A, Stilger M, Woods WS, Gosstola N, Gapinske M, Zeballos A, Moore H, Maslov S, Gaj T, Perez-Pinera P. Miskalis A, et al. Among authors: gaj t. Nat Commun. 2024 Nov 28;15(1):10354. doi: 10.1038/s41467-024-54529-y. Nat Commun. 2024. PMID: 39609418 Free PMC article.

3

In vivo CRISPR base editing for treatment of Huntington's disease.

Shirguppe S, Gapinske M, Swami D, Gosstola N, Acharya P, Miskalis A, Joulani D, Szkwarek MG, Bhattacharjee A, Elias G, Stilger M, Winter J, Woods WS, Anand D, Lim CKW, Gaj T, Perez-Pinera P. Shirguppe S, et al. Among authors: gaj t. bioRxiv [Preprint]. 2024 Jul 6:2024.07.05.602282. doi: 10.1101/2024.07.05.602282. bioRxiv. 2024. PMID: 39005280 Free PMC article. Preprint.

4

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

Miskalis A, Shirguppe S, Winter J, Elias G, Swami D, Nambiar A, Stilger M, Woods WS, Gosstola N, Gapinske M, Zeballos A, Moore H, Maslov S, Gaj T, Perez-Pinera P. Miskalis A, et al. Among authors: gaj t. bioRxiv [Preprint]. 2024 Apr 2:2024.04.01.587650. doi: 10.1101/2024.04.01.587650. bioRxiv. 2024. Update in: Nat Commun. 2024 Nov 28;15(1):10354. doi: 10.1038/s41467-024-54529-y PMID: 38883727 Free PMC article. Updated. Preprint.

5

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

McCallister TX, Lim CKW, Terpstra WM, Alejandra Zeballos C M, Zhang S, Powell JE, Gaj T. McCallister TX, et al. Among authors: gaj t. bioRxiv [Preprint]. 2023 Dec 12:2023.12.12.571328. doi: 10.1101/2023.12.12.571328. bioRxiv. 2023. PMID: 38168370 Free PMC article. Preprint.

6

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

Zeballos C MA, Moore HJ, Smith TJ, Powell JE, Ahsan NS, Zhang S, Gaj T. Zeballos C MA, et al. Among authors: gaj t. Nat Commun. 2023 Oct 14;14(1):6492. doi: 10.1038/s41467-023-42147-z. Nat Commun. 2023. PMID: 37838698 Free PMC article.

7

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

Zeballos C MA, Moore HJ, Smith TJ, Powell JE, Ahsan NS, Zhang S, Gaj T. Zeballos C MA, et al. Among authors: gaj t. bioRxiv [Preprint]. 2023 Aug 26:2023.04.07.536072. doi: 10.1101/2023.04.07.536072. bioRxiv. 2023. Update in: Nat Commun. 2023 Oct 14;14(1):6492. doi: 10.1038/s41467-023-42147-z PMID: 37066174 Free PMC article. Updated. Preprint.

8

Delivering Base Editors In Vivo by Adeno-Associated Virus Vectors.

Lim CKW, Miskalis AJ, Perez-Pinera P, Gaj T. Lim CKW, et al. Among authors: gaj t. Methods Mol Biol. 2023;2606:135-158. doi: 10.1007/978-1-0716-2879-9_11. Methods Mol Biol. 2023. PMID: 36592313

9

CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genes.

Lim CKW, McCallister TX, Saporito-Magriña C, McPheron GD, Krishnan R, Zeballos C MA, Powell JE, Clark LV, Perez-Pinera P, Gaj T. Lim CKW, et al. Among authors: gaj t. Mol Ther. 2022 Dec 7;30(12):3619-3631. doi: 10.1016/j.ymthe.2022.08.008. Epub 2022 Aug 13. Mol Ther. 2022. PMID: 35965414 Free PMC article.

10

Targeted gene silencing in the nervous system with CRISPR-Cas13.

Powell JE, Lim CKW, Krishnan R, McCallister TX, Saporito-Magriña C, Zeballos MA, McPheron GD, Gaj T. Powell JE, et al. Among authors: gaj t. Sci Adv. 2022 Jan 21;8(3):eabk2485. doi: 10.1126/sciadv.abk2485. Epub 2022 Jan 19. Sci Adv. 2022. PMID: 35044815 Free PMC article.

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