Maria Montiel

maria montielMaria F. Montiel
Research Associate
Email Maria


Research Interests:

In recent years, strategies to correct genetic mutations by modifying DNA and RNA molecules have received increasing attention. My project has been based on engineering a site-directed RNA editing strategy to use it as a tool to correct specific mistakes within mRNAs.

RNA editing is an enzymatic process that performs adenosine (A) to inosine (I) changes within RNAs. This process is catalyzed by adenosine deaminases that act on RNA (ADARs). Inosine is structurally similar to guanosine (G), and an A-to-I change is interpreted as an A-to-G change by ribosomes and other biological processes. As a result of this mechanism, protein function may be altered, particularly if editing occurs within mRNA coding regions.

In the laboratory we have engineered an “editase” that can direct the editing process to a specific adenosine of our choosing (for a description of our strategy, see R. Reenan New Engl J Med 370:172-174). Using this strategy, we have been able to correct a premature termination codon (PTC) mutation in an eGFP fluorescent reporter with a 70 percent of editing efficiency. At present, we are testing our strategy in several mutations that cause cystic fibrosis and can make corrections within cells at efficiencies ranging from 20 percent to 80 percent.