Downstream purification of RNA therapeutics

Gene therapy is an expanding field that shows promise in the long term treatment of many genetic disorders. CRISPR-Cas9 technology is one way of editing the existing genome when trying to treat genetic disorder, or engineering genetic therapeutics. One area of interest is RNA therapeutics based on this technology. The Zydney and Salis lab groups at Penn State have been producing Cas9 protein using the CSL Behring Fermentation Facility. These proteins are then coupled to guide RNA for the specific gene therapy application. One of the challenges is that there is often residual guide RNA that needs to be removed after the coupling reaction. This project will explore the potential for using ultrafiltration systems for the separation of the protein-RNA complex from the free RNA. This work will focus on determining the effect that factors such as filtrate flux, pore size, product size, and surface chemistry have on RNA transmission.

Faculty Supervisor:

David Latulippe

Student:

Partner:

Pennsylvania State University

Discipline:

Engineering

Sector:

Education

University:

McMaster University

Program: