RNA Clinical Discovery

RNA Clinical Discovery (750 x 500)The Clinical Discovery Group within the RNA Bioscience Initiative seeks to develop new RNA therapeutics for disease diagnosis and treatment.

RNAs are informative readouts of disease status: the presence or absence of particular RNAs and isoforms can be specific to particular pathologic conditions, e.g., infection, cancer, and ischemia. Because the transcriptomes of normal and diseased cell types are complex and often poorly characterized, the potential utility of RNAs as biomarkers has not been fully exploited. The advent of new high throughput technologies, especially massively parallel single-cell RNA sequencing, empowers our researchers to fill this void.

An exceptional feature of RNA therapeutics is their ability to target any RNA via complementary sequence, enabling therapies for many diseases. RNA therapies are becoming increasingly common mainstream clinical tools. The RBI recently partnered with Children's Hospital Colorado to develop antisense oligonucleotide therapies for pediatric neurogenetic diseases.

Recent RNA-based vaccines developed by Pfizer and Moderna for SARS-CoV-2 have demonstrated remarkable potency, possibly ushering in a new era of RNA-based immunological tools.

The Denver/Boulder area is a major hub of RNA biotechnology. Establishment of partnerships with local companies is a priority for the RBI Clinical Discovery Group.

Faculty Participants

Tom Anchordoquy, PhDtom.anchordoquy@cuanschutz.eduDevelopment of synthetic delivery systems for use in nucleic acids-based therapies 
Matthew Burchill, PhDmatthew.burchill@cuanschutz.eduDisease therapeutics targeting lymphatic endothelial cells
Heide Ford, PhDheide.ford@cuanschutz.eduSmall molecule drug design to inhibit Six1/Eya transcriptional complex in tumors
Elena Hsieh, MDelena.hsieh@cuanzchutz.eduDevelopment of improved diagnostics and therapeutic treatments for immune system disorders
Peter Kabos, MDpeter.kabos@cuanschutz.eduImproved diagnostics and therapeutics to address anti-endocrine therapy resistant breast cancers
Jeffrey Kieft, PhDjeffrey.kieft@cuanschutz.eduRNAs as targets for small-molecule or within mRNA-based therapies (with industry partners)
Martin McCarter, MDmartin.mccarter@cuanschutz.eduTherapeutics to inhibit BCL2 family members in melanoma 
Karen Moulton, MDkaren.moulton@cuanschutz.eduModulation of PTEN to treat pathological vascular fibrosis
J. David Port, PhDdavid.port@cuanschutz.eduMicroRNAs in heart failure, cardiac transplantation and myocardial recovery
Yiqun Shellman, PhDyiqun.shellman@cuanschutz.edExploration of miR-26a replacement as therapy for treating melanoma
Matt Taylor, MD, PhDmatthew.taylor@cuanschutz.eduAntisense oligonucleotide mediated exon skipping (AON) to prevent early termination of titin as a treatment for heart failure
Rui Zhao, PhDrui.zhao@cuanschutz.eduSmall molecule drug design to inhibit Six1/Eya transcriptional complex in tumors