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RNA Bioscience Initiative Summary

Jay Hesselberth
February 11, 2020

RNA Bioscience Initiative Summary

Overview of RBI Accomplishments and Plans

Jay Hesselberth

February 11, 2020
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  1. What is RNA? Tom Cech’s studies of RNA led to

    the first CU Nobel Prize DNA RNA PROTEIN Many other Nobel Prizes from surprising discoveries in RNA biology (RNAi, ribosome, telomerase, genetic code, reverse transcriptase, etc.) Larry Gold developed RNA aptamers and founded many biotech spinoffs Norm Pace founded “microbiome” analysis: ribosomal RNA sequencing Jennifer Doudna et al discovered CRISPR/Cas9 “gene editing machines” guided to their targets by RNA RNA is the target of antisense oligonucleotide therapies for neurodegenerative diseases 2019 Breakthrough Prize to Adrian Krainer (Dean’s Distinguished on March 10)
  2. RBI Accomplishments 1. National recognition as a center of excellence

    in RNA biology. • 36 RBI seminars, 14 RNA Clubs, Rocky Mountain RNA Symposium 2. Established a robust pipeline for training RNA biologists. • Undergraduate > Graduate > Postdoctoral • Major impact on recent renewal of NIGMS Molecular Biology T32 (50 slots over 5 years) 3. Cluster hire of 5 faculty with expertise across RNA biology. • Each with R01 funding or foundation & pilot grants 4. Developed a postdoctoral program for domain-specific bioinformatics support. 5. Disseminated new technologies on campus via pilot funding. • 50 publications on PubMed supported by RBI • 1 provisional patent and 3 disclosures on new single-cell technologies
  3. RBI Sustainability • The RBI Informatics Fellows have fundamentally reshaped

    campus bioinformatics and provide a model for future bioinformatics efforts. • Completed 58/118 projects with 84 investigators. Co-authored 10 publications (7 in prep) and 17 NIH grant applications. • Training and disseminating talent across multiple disciplines with a focus in RNA. • Funded via focused collaborations and T32 / F32 fellowship support. • Future plans: RNA-focused R25 and T32 • RBI developed significant educational offerings in RNA biology and informatics • Summer internship for RNA research = many new grad school applicants interested in RNA • Informatics Fellows host single-cell mRNA sequencing workshop, R/RStudio course, weekly office hours • Major impact on Molecular Biology T32 renewal • RBI initiated clinical & translational collaborations with long-term potential • Pediatric neuro-oncology (single-cell compendia of brain tumors, 2 papers submitted & in prep) • Pediatric neuro-genetics (RBI-funded patient record review. milasen & ASO development) • Immunology (single-cell technologies, 2 disclosures filed) • Future plans: multi-PI R01s and Program Projects • Hiring Grants & Development Coordinator to help create large research and training grants • Educate campus community about RNA, facilitate new interactions & collaborations, interact with Office of Advancement
  4. Was the RBI transformative? • $20 million = $10m program

    project + $10m faculty hires • Our investment in faculty hires is paying off • How does NIH measure the success of a Program Project? • RBI publication (50+) and patent (>1) output is already equivalent to typical P01 • RBI also provides robust training programs, bioinformatic analysis infrastructure, new collaborations. • Major impact on awareness of RNA analysis in campus research groups • National recognition has led to enhanced recruitment 1 10 100 1000 1 10 100 Total cost (USD, millions) Number of publications institute AG AI CA HD HL P01 n=434, <= 5 year duration, ending after 2010 NIH Program Project output