Assistant Professor of Internal Medicine
- Cardiovascular Medicine
Web: PubMed publications
PhD, Molecular Physiology & Biophysics, The University of Iowa
Post Doctorate, The University of Iowa
My laboratory studies post-transcriptional responses in diseased and developing heart and brain using viral vectors, mouse models, human tissues, and cell culture systems. Our goal is to identify disease-related RNA-binding proteins (RBPs) and microRNAs (miRNAs) and determine their regulatory targets using computational and high-throughput biochemical means (HITS-CLIP, CLIP-seq). RBPs and miRNAs are key mediators of post-transcriptional responses, and modulation of their activities provides attractive means to manipulate gene expression for therapeutic benefit.
Another major aspect of my laboratory’s research involves determining how human genetic variations contribute to disease by altering post-transcriptional regulatory mechanisms. This work aims to generate valuable resources (e.g. miRNA target interactomes in human tissues) to accelerate our understanding of miRNA and RBP functions in heart and brain and facilitate the translation of available and ongoing genetic studies towards novel or refined disease mechanisms and therapies.
In addition to this, we are also investigating the functions of several novel genes, including long non-coding RNAs, in heart and brain. These projects incorporate a breadth of techniques, including viral-based (AAV) overexpression and inhibition (RNAi) of genes in vivo, generation and characterization of CRISPR-derived knockout mice, and gene transfer and functional assays in human iPS-derived cells.
Overall, my research program is balanced in basic and translational studies, wet-lab and computational methods, and resource- and hypothesis-driven research. This framework promotes multi-disciplinary and collaborative science, offering an excellent environment to foster the growth of current and future trainees.
Transcriptome-wide discovery of miRNA binding sites in human brain.
siSPOTR: a tool for designing highly specific and potent siRNAs in human and mouse.
Nucleic Acids Research.
Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo.
Non-allele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington’s disease mice.
Minimizing variables among hairpin-based RNAi vectors reveals improved potency of shRNAs.
Mas Monteys A,
Artificial microRNAs mitigate short hairpin RNA-mediated toxicity in the brain: Implications for the therapeutic development of RNA interference.
Proceedings of the National Academy of Sciences USA .
Date Last Modified: 04/12/2016 -