Nucleic acids are the building blocks of DNA and RNA. Oligonucleotides are chemically synthesized short DNA or RNA molecules that can be designed and used as drugs when targeting specific regions of DNA or RNA to regulate or control the transcription or translation of DNA or mRNA. Antisense oligonucleotides (ASOs) are single strands of DNA or RNA that are complementary to a chosen sequence and often used to prevent the translation of an mRNA into protein, however, ASOs can also be designed to specific targets that can increase the expression of a gene.
Friedreich’s Ataxia Research Alliance, FARA is funding research in Dr. David Corey’s lab at UT Southwestern where he has been testing ASOs and other oligonucleotides called gapmers that target the GAA repeat in the frataxin gene or mRNA as a way to activate or produce more frataxin protein. FARA is also funding research in Dr. Jonathan Watt’s lab at the University of Massachusetts where they are improving the chemistry of oligonucleotides so that they are able to reach more specific regions in the brain and the periphery (eg., the heart). In addition, Watt’s lab is trying to identify novel (non-GAA repeat) targets within the frataxin gene where oligonucleotides could be designed to activate frataxin expression.