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. The vector genom...

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Publicado no:iScience
Main Authors: Krooss, Simon Alexander, Dai, Zhen, Schmidt, Florian, Rovai, Alice, Fakhiri, Julia, Dhingra, Akshay, Yuan, Qinggong, Yang, Taihua, Balakrishnan, Asha, Steinbrück, Lars, Srivaratharajan, Sangar, Manns, Michael Peter, Schambach, Axel, Grimm, Dirk, Bohne, Jens, Sharma, Amar Deep, Büning, Hildegard, Ott, Michael
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6941859/
https://ncbi.nlm.nih.gov/pubmed/31887661
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.isci.2019.100764
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