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Site-directed RNA Editing by Engineered Human ADAR2

Efficient system to selectively re-code genetic information within mRNAs with potential to restore structure/function of defective proteins

Background

Many diseases like cystic fibrosis, β-thalassemia, and several disabling syndromes can be caused by a single point mutation within an open reading frame. Single base substitutions can lead to a Premature Termination Codon (PTC) or an amino acid substitution, resulting in the truncation of a protein or loss of its function, thus producing the defective phenotype.

DNA editing tools like CRISPR-Cas9 systems have captivated the attention for the development of therapeutics for a wide variety of diseases. It offers a life-long solution to chronic diseases by permanent genetic modifications. However, off target events are major concerns because cellular function may be disrupted permanently. RNA editing-based therapies can make transient changes in the genetic information, eliminating the concern of making

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