Cell-specific mRNA design yielding highly efficient and safe gene editing in human epithelia

Gene therapies have the potential to help many patients with currently untreatable, severe, and life-threatening diseases. A certain type of gene therapy may even provide a cure for patients with genetic diseases. A key component of these gene therapies often is RNA. However, we are still lacking a good understanding of how RNA should ideally be designed and manufactured, especially when striving for an application beyond vaccination. Here, we are particularly interested in the lung as the target organ as several lung diseases would potentially benefit from an effective and safe gene therapy – many of them are poorly treatable to date.
Also, we currently do not know how RNA should be designed to exert especially powerful effects in specific cell types while maintaining good biocompatibility. Hence, in this project, we aim to understand the impact of RNA design and modifications when targeting the human lungs. We will generate a variety of RNA variants, assess the effect of these different structures on the efficacy and tolerability in the lungs, and develop criteria for other researchers how to effectively design safe and efficient RNA molecules for lung applications…

Faculty Supervisor:

Eric Jan;Sarah Hedtrich

Student:

Partner:

Providence Health Care

Discipline:

Life Sciences

Sector:

Health and Related Sciences & Technology; Professional, scientific and technical services

University:

The University of British Columbia

Program: