Loading...

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...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i: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
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2019
Fag:
Online adgang: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
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!