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...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:iScience
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2019
Témata:
Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky