APOBEC1 mutants for the development of new Base Editors with reduced RNA / DNA Off-targets

# Record card
Thematic areas
Health & Biotech
Health & Biotech / Bio-medicals
Health & Biotech / New therapies
Health & Biotech / Diagnostic kits

The development of genome editing tools has revolutionized the way we think and deal with genetics. The use of Cas9 or its variants allows modifications of specific sites in the human genome by inducing deletions and insertions in a more or less controlled way. In recent years, a new class of tools for genome editing has emerged: the base editors (BE), which result from the fusion of a modified Cas9, which serves to direct the BE to the target, and an active deaminase acting on the DNA, which mediates the C> T or A> G editing. These molecules allow the modification of single bases in a specific DNA region without inducing double strand breaks, and therefore could be safer than using Cas9 alone. As for C> T editing, the most commonly used deaminase is rat APOBEC1, a powerful DNA mutator that acts physiologically on RNA.

Business fields
Type of innovation
Product / process innovation in integration with an already existing technology
Description of innovative features / Competitive advantages

Despite their potential, several studies have shown that Base Editor expression in human cells causes off-target mutations in both RNA and DNA. To limit these mutations, we have used bacterial screening and assays in human cells to develop mutants of the APOBEC1 deaminase with substantially lower levels of off-target mutations both on RNA and on DNA. Their incorporation within base editors presents a considerably reduced number of off-target mutations both on RNA and DNA. These new base editors we have developed can be used to induce a safer genomic editing.

Reference market
Incremental innovation
Development stage
Product/process/service/technology optimization
Patented technology


Publication of technology
In publication phase
Technology validation/demonstration
Internal validation
Market positioning
Partner required
Public research center/university
Private research center
Cooperation in national /european / international project

For more information and/or to be put in contact with the Research Team, please contact the Project Manager:

Barbara Angelini - Project Manager
CNR - Unità Valorizzazione della Ricerca
Phone number 06.49932415
E-mail barbara.angelini@cnr.it