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Ex Vivo/In vivo gene editing in Hepatocytes using “all-in-one” CRISPR-adeno-associated virus vectors with a self-linearizing repair template

Adeno-associated virus (AAV)-based vectors are considered efficient and safe gene delivery systems in gene therapy. We combined two guide RNA genes, Cas9, and a self-linearizing repair template in one vector (AIO-SL) to correct fumarylacetoacetate hydrolase (FAH) deficiency in mice.

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Bibliographic Details
Main Authors: Krooss, Simon (Author) , Dai, Zhen (Author) , Schmidt, Florian (Author) , Rovai, Alice (Author) , Fakhiri, Julia (Author) , Dhingra, Akshay (Author) , Yuan, Qinggong (Author) , Yang, Taihua (Author) , Balakrishnan, Asha (Author) , Steinbrück, Lars (Author) , Srivaratharajan, Sangar (Author) , Manns, Michael Peter (Author) , Schambach, Axel (Author) , Grimm, Dirk (Author) , Bohne, Jens (Author) , Sharma, Amar Deep (Author) , Büning, Hildegard (Author) , Ott, Michael (Author)
Format: Article (Journal)
Language:English
Published: 2020
In: iScience
Year: 2019, Volume: 23, Issue: 1
ISSN:2589-0042
DOI:10.1016/j.isci.2019.100764
Online Access:Resolving-System, lizenzpflichtig, Volltext: https://doi.org/10.1016/j.isci.2019.100764
Verlag, lizenzpflichtig, Volltext: http://www.sciencedirect.com/science/article/pii/S2589004219305097
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Author Notes:Simon Alexander Krooss, Zhen Dai, Florian Schmidt, Alice Rovai, Julia Fakhiri, Akshay Dhingra, Qinggong Yuan, Taihua Yang, Asha Balakrishnan, Lars Steinbrück, Sangar Srivaratharajan, Michael Peter Manns, Axel Schambach, Dirk Grimm, Jens Bohne, Amar Deep Sharma, Hildegard Büning, Michael Ott

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