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Adeno-associated viral vector resource for the RNA-targeting Cas13d: A comparison of high-fidelity variants, DjCas13d and hfCas13d.

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第一作者： Franklin,Back

第一单位： Department of Neuroscience and Experimental Therapeutics, Penn State College of Medicine, Hershey, PA 17033-0850, USA.

作者： Franklin,Back ^[1] ; Alfredo,Sandoval ^[2] ; Lily M,Vu ^[3] ; Veronica M,Hong ^[3] ; Amulya,Bhaskara ^[3] ; Sierra R,Rodriguez ^[3] ; John T,O'Brien ^[3] ; Benedict J,Kolber ^[3] ; Sven,Kroener ^[3] ; Jonathan E,Ploski ^[1]

作者单位： Department of Neuroscience and Experimental Therapeutics, Penn State College of Medicine, Hershey, PA 17033-0850, USA. ^[1] The University of Texas Medical Branch, Galveston, TX 77555, USA. ^[2] Department of Neuroscience, School of Behavioral and Brain Sciences, The University of Texas at Dallas, Richardson, TX 75080, USA. ^[3]

关键词 AAV CRISPR/Cas Cas13 CasRx RNA degradation RfxCas13d collateral-cleavage highly-expressed transcripts protein expression transcriptome changes

DOI 10.1016/j.omtm.2025.101565

PMID 41020266

发布时间 2025-10-01

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Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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Adeno-associated viral vector resource for the RNA-targeting Cas13d: A comparison of high-fidelity variants, DjCas13d and hfCas13d.

Molecular therapy. Methods & clinical development

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Adeno-associated viral vector resource for the RNA-targeting Cas13d: A comparison of high-fidelity variants, DjCas13d and hfCas13d.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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