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Welcome to the Emeson Lab

The research interests of our laboratory focus on the cellular and molec­ular processes under­ly­ing neuronal com­mun­i­ca­tion in nor­mal and patho­phys­i­o­log­ic disease states. Spe­cif­ically, we are examining the molecular mechanisms involved in the editing of RNA transcripts encoding proteins critical for mammalian nervous system function. RNA edit­ing is a post-transcriptional modification in which specific adenosine residues in pre-mes­senger RNAs are converted to inosine (A-to-I editing) by double-strand­ed RNA-spe­cif­ic adeno­sine deaminases (ADARs). As a result of these deamina­tion events, the cod­ing poten­tial of RNAs can be subtly altered to change as little as a single amino acid residue in resultant products to generate protein isoforms with distinct functional proper­ties.

Programmable alterations of nucleic acid sequence offer significant therapeutic potential for a wide range of genetic disorders. In recent years, the RNA molecule has become one of the most promising targets for ther­apeutic inter­ven­tion and many RNA-based therapeutics have been developed recently. Despite the promise of CRISPR/Cas9-based ap­proach­es for the editing of genomic DNA, this paradigm has numerous disadvantages, including off-target and imprecise modifications, as well as a low efficiency rate in post-mitotic cell types such as neurons.

Our current research efforts focus upon an altern­ative approach for tar­get­ed gen­ome en­gin­­eer­ing using cellular processes norm­­al­ly in­volv­ed in the A-to-I editing of RNA tran­scripts. These studies involve the development of a systematic pipeline for the selection of ADAR-recruiting RNAs (arRNAs) that will promote efficient, site-selective adenosine deamination to repair common mutations associated with neurological disorders such as Parkinson’s Disease and Rett Syndrome.

It is anticipated that these studies will allow the pre-clinical development of therapeutic arRNAs for the  repair of numerous genomic variants leading to disease in human­ized mouse models and affected pati­ents.