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Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins.
Nat Commun. 2023 Oct 14;14(1):6492. doi: 10.1038/s41467-023-42147-z.
Nat Commun. 2023.
PMID: 37838698
Free PMC article.
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.
bioRxiv [Preprint]. 2023 Aug 26:2023.04.07.536072. doi: 10.1101/2023.04.07.536072.
bioRxiv. 2023.
PMID: 37066174
Free PMC article.
Updated.
Preprint.
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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: zeballos c ma.
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.
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Next-Generation CRISPR Technologies and Their Applications in Gene and Cell Therapy.
Zeballos C MA, Gaj T.
Zeballos C MA, et al.
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.
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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: zeballos c ma.
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.
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