MSTP in PhD Training
Current advisor: Timothy M. Miller, MD, PhD
Undergraduate university: University of Alabama-Birmingham, 2018
Enrollment year: 2018
Using antisense oligonucleotides to alter protein expression in neurological disease
In the lab of Dr. Tim Miller, I study the regulation of RNA and strategies to alter RNA processing and translation. On any particular transcript, the untranslated regions (UTRs) that flank the coding sequence contain recognition sites for a number of trans-acting elements. Their interactions with RNA are essential for normal processing and for determining RNA stability and translation efficiency. Each gene contains unique UTRs, so approaches that target these regions might afford regulation in a gene transcript-specific manner. Our lab specializes in strategies using antisense oligonucleotide (ASOs), which are powerful investigational tools with proven translational potential. ASOs are short DNA or RNA oligonucleotides that bind to a particular transcript with high sequence-specificity. Depending on their design, they might influence processing of the transcript, such as altering splicing, or degrade a transcript by recruiting RNases. Importantly, they have the ability to sterically block interactions with trans-acting elements or even modify secondary structures, which often facilitate these interactions. Focusing on genes implicated in neurodegenerative disease, I study whether targeting UTRs using ASOs can be used to alter mRNA stability or translation.
Boros BD, Schoch KM, Kreple CJ, Miller TM. 2022 Antisense Oligonucleotides for the Study and Treatment of ALS. Neurotherapeutics, ():Epub ahead of print.