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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:iScience
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Elsevier 2019
Aiheet:
Article
Linkit: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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