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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Enregistré dans:
Détails bibliographiques
Publié dans:iScience
Auteurs principaux: 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
Format: Artigo
Langue:Inglês
Publié: Elsevier 2019
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Article
Accès en ligne: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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