Bioengineering advances have now made it feasible for precision gene editing directly in patient tissues with the goal of not only treatment, but of a cure. In this project, the priority is genetic disorders that inflict the eye, specifically the retinal pigmented epithelium (RPE) that surrounds the retina.
Existing state-of-the-art approaches to edit cells within human tissues have significant limitations both with respect to safety and efficacy. The nanomedicine approach of this project is anticipated to overcome these limitations and will set the foundation for a new paradigm in developing genomic medicine.
This project will use advances in biomaterials to generate nonviral, synthetic nanocarriers of CRISPR-Cas9 gene editing machinery for targeted delivery to the RPE. Such research would generally expand the types of tissues that could be edited and hence the spectrum of disease where genomic medicine could have an impact. This hypothesis will be tested to precisely edit pathogenic point mutations in vivo within transgenic mouse disease models and in patient-derived, induced pluripotent stem cell (iPSC)-based disease models.
PRINCIPAL INVESTIGATOR- Krishanu Saha
Assistant Professor of Biomedical Engineering at the Wisconsin Institute for Discovery
- Shaoqin Sarah Gong
Professor of Biomedical Engineering at the Wisconsin Institute for Discovery - Bikash Pattnaik
Assistant Professor of Pediatrics - David Gamm
Associate Professor of Ophthalmology and Visual Sciences and Director of the McPherson Eye Research Institute
May 3, 2018 By Natasha Kassulke
Seventeen innovative projects on such diverse topics as personalizing diabetes prevention and treatment, transforming wood into a renewable electronic material, improving outcomes for incarcerated parents and their children, and advancing the frontier of fusion energy sciences have been chosen to join the UW2020: WARF Discovery Initiative grant cohort.
One UW2020 project will will focus on fostering positive family interactions and relationships between incarcerated parents and their children through visit coaching in the jail and home, and offering in-home video visits via One Laptop per Child. PHOTO: COURTESY OF JULIE POEHLMANN-TYNAN
These projects, with an average award amount of $432,442, were among 111 proposals submitted from across the UW–Madison campus. The initiative is funded by the Office of the Vice Chancellor for Research and Graduate Education and the Wisconsin Alumni Research Foundation.
This is the fourth round of UW2020 project selections and brings the total number of UW2020 projects to 66 since the first round awards were announced in 2016. This round includes six infrastructure projects and 11 research projects that cross multiple divisions on campus. Funded projects include 125 faculty and academic staff investigators on the project teams from 10 schools and colleges.
Another project has the potential to radically improve existing disaster management practices, and save lives and resources by providing advanced preparedness and response strategies. PHOTO: COURTESY OF PAUL BLOCK
The projects were reviewed by faculty across the university, ultimately involving 102 reviewers. The UW2020 Council, a group of 17 faculty from all divisions of the university, evaluated the merits of each project based on the reviews and their potential for making significant contributions to their field of study.
The goal of UW2020 is to stimulate and support cutting-edge, highly innovative and groundbreaking research at UW–Madison and to support the acquisition of shared instruments or equipment that will foster significant advances in research.
Immunofluorescence image of a paraffin-embedded pancreas section stained with antibodies. Allowing for the precision treatment of individuals with type 1 and type 2 diabetes, is a key goal of this UW2020 project. PHOTO: COURTESY OF MICHAEL SCHAID, KATHRYN CARBAJAL AND MICHELLE KIMPLE
“While previous rounds of UW2020 projects are now maturing and realizing their potential, we are excited to infuse the initiative with a new class of inspiring and novel projects that continue to showcase UW–Madison’s highly competitive and forward-thinking world-class faculty and staff researchers,” says Norman Drinkwater, interim vice chancellor for research and graduate education.
“UW–Madison remains very grateful to WARF for continuing to support and partner with us on this initiative,” he says.
The Graduate School also supplies direct support for some research assistants.
A list of projects and their principal investigators can be found below, but visit the UW2020 website to find project descriptions and more information.
Research ProjectsAdvancing CRISPR-mediated Genome Editing Technology at UW–Madison to Model Human Disease
Anjon Audhya, Associate Professor of Biomolecular Chemistry
All-Optical Electrophysiology-Electrophysiology without Electrodes
Baron Chanda, Associate Professor of Neuroscience
Building a Translational Research Pipeline to Personalize Diabetes Prevention and Treatment
Elizabeth Cox, Associate Professor of Pediatrics
Communication Ecologies, Political Contention, and Democratic Crisis
Lewis Friedland, Professor of Journalism and Mass Communication
Development of a Forecast-based Flood and Health Risk Management System to Support Advanced Disaster Preparedness
Paul Block, Assistant Professor of Civil and Environmental Engineering
Gene Editing Nanomedicines to Correct Pathogenic Mutations in Retinal Pigmented Epithelium
Krishanu Saha, Assistant Professor of Biomedical Engineering at the Wisconsin Institute for Discovery
Improving Outcomes for Incarcerated Parents and their Children through Enhanced Jail Visits
Julie Poehlmann-Tynan, Professor of Human Ecology and Affiliate of the Institute for Research on Poverty, the Center for Child and Family Well-being and the Center for Healthy Minds
Instrument Development to Study the Highest Energy Photons in the Universe
Justin Vandenbroucke, Assistant Professor of Physics
Maternal Breathing Dysfunction during Pregnancy Increases Risk for Psychiatric Disorders in Her Offspring: A Paradigm-Shifting Concept
Tracy Baker, Associate Professor of Comparative Biosciences
Toxoplasma Infection and Brain Function
Meyer Jackson, Professor of Neuroscience
Transforming Wood into a Green, Renewable Electronic Material
Michael Arnold, Professor of Materials Science and Engineering
Accelerating Lead Optimization to Clinical Application Using Microscale Thermophoresis to Quantify Molecular Interactions
Weiping Tang, Professor of Pharmaceutical Sciences
Acquisition of an Illumnina NovaSeq Next Generation DNA Sequencer for UW–Madison
Michael Sussman, Professor of Biochemistry and Director of the Biotechnology Center
Data Science Hub for UW–Madison
Brian Yandell, Professor of Statistics
Fusion Energy Research at the Next Frontier: Integrating Optimized Plasma Confinement with Interdisciplinary Science and Engineering
David Anderson, Professor of Electrical and Computer Engineering
State-of-the-Art, Reactive-Ion-Etching Instrument for Nanofabrication of Devices at UW–Madison
Luke Mawst, Professor of Electrical and Computer Engineering
The LandLab Institute: A Place for Ecological Solutions to Land Use Problems
Randall Jackson, Professor of Agronomy