Gaj T - Search Results

1

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.

2

Treatment of a Mouse Model of ALS by In Vivo Base Editing.

Lim CKW, Gapinske M, Brooks AK, Woods WS, Powell JE, Zeballos C MA, Winter J, Perez-Pinera P, Gaj T. Lim CKW, et al. Among authors: gaj t. Mol Ther. 2020 Apr 8;28(4):1177-1189. doi: 10.1016/j.ymthe.2020.01.005. Epub 2020 Jan 14. Mol Ther. 2020. PMID: 31991108 Free PMC article.

3

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

Zeballos C MA, Gaj T. Zeballos C MA, et al. Among authors: gaj t. Trends Biotechnol. 2021 Jul;39(7):692-705. doi: 10.1016/j.tibtech.2020.10.010. Trends Biotechnol. 2021. PMID: 33277043 Free PMC article. Review.

4

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.

5

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.

6

Innovations in CRISPR technology.

Brooks AK, Gaj T. Brooks AK, et al. Among authors: gaj t. Curr Opin Biotechnol. 2018 Aug;52:95-101. doi: 10.1016/j.copbio.2018.03.007. Epub 2018 Apr 5. Curr Opin Biotechnol. 2018. PMID: 29626736 Review.

7

CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model.

Ekman FK, Ojala DS, Adil MM, Lopez PA, Schaffer DV, Gaj T. Ekman FK, et al. Among authors: gaj t. Mol Ther Nucleic Acids. 2019 Sep 6;17:829-839. doi: 10.1016/j.omtn.2019.07.009. Epub 2019 Jul 26. Mol Ther Nucleic Acids. 2019. PMID: 31465962 Free PMC article.

8

The continuously evolving CRISPR barcoding toolbox.

Gaj T, Perez-Pinera P. Gaj T, et al. Genome Biol. 2018 Sep 25;19(1):143. doi: 10.1186/s13059-018-1541-y. Genome Biol. 2018. PMID: 30253789 Free PMC article.

9

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins.

Gaj T, Guo J, Kato Y, Sirk SJ, Barbas CF 3rd. Gaj T, et al. Nat Methods. 2012 Jul 1;9(8):805-7. doi: 10.1038/nmeth.2030. Nat Methods. 2012. PMID: 22751204 Free PMC article.

10

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

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