Title:Artificial RNA Editing with ADAR for Gene Therapy
Volume: 20
Issue: 1
Author(s): Sonali Bhakta and Toshifumi Tsukahara*
Affiliation:
- Area of Bioscience and Biotechnology, School of Materials Science, Japan Advanced Institute of Science and Technology, 1-1 Asahidai, Nomicity, Ishikawa, 923-1292,Japan
Keywords:
RNA editing, ADAR, mutation, MS2 system, enzyme application, genetic code restoration.
Abstract: Editing mutated genes is a potential way for the treatment of genetic diseases. G-to-A mutations
are common in mammals and can be treated by adenosine-to-inosine (A-to-I) editing, a type of
substitutional RNA editing. The molecular mechanism of A-to-I editing involves the hydrolytic deamination
of adenosine to an inosine base; this reaction is mediated by RNA-specific deaminases, adenosine
deaminases acting on RNA (ADARs), family protein. Here, we review recent findings regarding
the application of ADARs to restoring the genetic code along with different approaches involved in
the process of artificial RNA editing by ADAR. We have also addressed comparative studies of various
isoforms of ADARs. Therefore, we will try to provide a detailed overview of the artificial RNA
editing and the role of ADAR with a focus on the enzymatic site directed A-to-I editing.