In alignment with The National Center for Advancing Translational Sciences’ (NCATS), the Utah Clinical & Translational Science Institute’s (CTSI) TIP program will support translational science projects that focus “on understanding a scientific or operational principle underlying a step of the translational process with the goal of developing generalizable principles to accelerate translational research.”
FY '23 Awardees
- Dr. Anne Thackeray, PhD, PT, MPH
- Title: Establishing a Functional Performance Index
- Project Narrative: Physical function is an essential component for quality of life. Functional performance tests measure function by directly observing how well certain activities can be performed. This can include measures of gait speed, stair climbing, or gripping power. These measures are often on different scales and difficult to compare across populations. This project will establish a scale that provides a single estimate of functional performance. This will help use compare treatments and populations. We will further examine how a person’s perception of their function, or self-report, is related to functional performance. From this we will understand if both functional performance and self-reported function should be measured, or if one measure will suffice.
- Dr. Hilary Coon, PhD
- Title: Community Consultation on Research Use of Autopsy Blood Spots in Utah
- Project Narrative: This project will develop guidelines for conducting community-informed research. As a model for this development, we will explore and implement aspects of community perceptions and opinions regarding research uses of an archived resource of ~30,000 blood spots from decedents obtained from the Utah State Office of the Medical Examiner. Preliminary linking to longitudinal medical records data indicates the presence of many diagnoses (e.g., diabetes, heart disease, etc.) among decedents in this resource, suggesting feasibility of genetic discovery across multiple areas of medical research. This study will explore optimal ways to conduct research using this resource through input from community leaders. Issues to be addressed will include risk perceptions, sample/data access, research design, limitations on sample use, and appropriate communication of results to community members. Community leaders will also help guide the selection of a common health condition, and will help direct the development and editing of a proposal focused on this condition that will include community-informed hypotheses, study design aspects, and return of results methods. Finally, our process of community engagement and community-informed proposal design will allow us to create more general guidelines for the implementation of community-based research. These guidelines will be broadly disseminated, and will serve as a practical guide for designing future community-informed research studies.
- Dr. Jun Yang, PhD
- Title: A pilot study on gene therapy for retinal degeneration
- Project Narrative: Gene replacement therapy for monogenic diseases is inapplicable to deliver large genes into targeted cells because of the package limits of common viral vectors and the low delivery efficiency of non-viral nanoparticles. In this proposal, we will test a new CRISPR/Cas9-mediated homology-independent exon replacement (HIER) strategy to correct mutations in a large gene USH2A, which are the leading cause of inherited deaf-blindness and are prevalent in autosomal recessive retinal degeneration.