Article
Optimized TAL effector nucleases (TALENs) for use in treatment of sickle cell disease.
Molecular bioSystems - 1 Apr 2012
Sun Ning, Liang Jing, Abil Zhanar, Zhao Huimin
Abstract excerpt
TAL effector nucleases (TALENs) represent a new class of artificial nucleases capable of cleaving long, specific target DNA sequences in vivo and are powerful tools for genome editing with potential therapeutic applications. Here we report a pair of custom-designed TALENs for targeted genetic correction of the sickle cell disease mutation in human cells, which represents an example of engineered TALENs capable of...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
