A strong micro-promoter for AAV

Case ID:
2700
Web Published:
10/9/2020
Description:

Summary

Viral mediated transgene delivery often utilizes specialized promoters that drive ubiquitous expression; however, these promoters are large and limit the amount of remaining space in AAV vectors for the transgene. The laboratory of Dr. Markus Grompe at OHSU has identified a novel promoter, which drives strong expression in multiple cell types and is roughly one tenth the size of currently used promoters, thereby allowing for delivery of larger transgenes.

 

Technology Overview

Promoters such as CMV and CAG are used to drive strong ubiquitous expression of transgenes, but these promoters take up a large portion (~1 kb) of the available packaging space (4.8 kb) within AAV vectors. Oregon Health & Science University (OHSU) researchers have identified a novel ubiquitous promoter that is a fraction of the size of currently used promoters at only 84 bp. This promoter drives high expression, comparable to CMV and CAG promoters, in multiple mammalian cell types including human pancreatic endocrine cells and primary hepatocytes in vitro, and murine hepatocytes and neurons in vivo. Experiments to validate in vivo expression in other cell types is ongoing. The considerable reduction in promoter size frees up valuable space for larger transgenes to be delivered by AAV vectors. In practical terms, switching from the traditional promoters to this micro-promoter version increases the available size for the transgene from 3.7 kb to 4.6 kb in a single stranded AAV and from 1.1 kb to 2 kb for self-complementary AAV. Utilization of this novel micro-promoter will allow for AAV delivery of a wider variety of transgenes to facilitate basic research and therapeutic development.

 

Publication

None

 

Intellectual Property

Patent application pending

 

Licensing Opportunity

Available for licensing and collaborative co-development

 

Patent Information:
For Information, Contact:
Travis Cook
Senior Technology Development Manager
Oregon Health & Science University
cooktr@ohsu.edu
Inventors:
Markus Grompe
Sunghee Chai
Keywords:
Biological Materials
Therapeutics
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