Article
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.
Molecular therapy : the journal of the American Society of Gene Therapy - 2 May 2018
Singh Kshitiz, Evens Hanneke, Nair Nisha, Rincón Melvin Y, Sarcar Shilpita, Samara-Kuko Ermira, Chuah Marinee K, VandenDriessche Thierry
Abstract excerpt
In vivo tissue-specific genome editing at the desired loci is still a challenge. Here, we report that AAV9-delivery of truncated guide RNAs (gRNAs) and Cas9 under the control of a computationally designed hepatocyte-specific promoter lead to liver-specific and sequence-specific targeting in the mouse factor IX (F9) gene. The efficiency of in vivo targeting was assessed by T7E1 assays, site-specific Sanger...
Topics
- Animals
- Base Sequence
- Binding Sites
- CRISPR-Cas Systems
- Computational Biology
- Dependovirus
- Factor IX
- Gene Editing
- Gene Targeting
- Genetic Vectors
- Hemophilia B