RNA Base Editing:Programmable Protein Machine

Abstract

RNA (ribonucleic acid) is a unique molecule which plays important and diverse roles in biology. In cells, RNA is ephemeral; it acts as a mediator, carrying genetic information from the nucleus to the protein-making machinery in the cytoplasm. A point mutation in RNA transcript could make the protein much shorter or larger than normal. Aberrant expression of RNAs effects the regulation of many cellular functions and gene networks. Most human genetic disorders such as sickle cell anaemia are due to just a single base-pair substitution, or by errors in RNA splicing, or its regulation. Precise editing of aberrant RNA provides a platform to correct the errors, without altering the genetic make-up of template DNA.