Polymers For Efficient Gene Delivery
ID U-5200
Category Therapeutics
Subcategory Biologics
Researchers
Brief Summary
Bio-reducible polymers for gene delivery with high transfection efficiency and low cytotoxicity.
Problem Statement
Both viral and non-viral delivery systems have been used for gene therapy, but non-viral vectors present a variety of advantages, including scalability, low immune response, flexible loading capacity, and stability. Widespread adoption of non-viral gene vectors, however, has been limited by toxicity and transfection efficiency concerns.
Technology Description
Multiple polymers for more efficient delivery of genetic material have been developed. The first is a novel arginine-conjugating bioreducible poly(amido amine) polymer that can degrade into nontoxic molecules in an intracellular environment. Mixing the polymer with PEG inhibits plasmid DNA condensation, improves biocompatibility, and enables in vivo applications. The second polymer is a poly(ethylamine) (PEI)-based gene delivery system, made through polymerization of cysteine and dendrimer branches. The polymer has a smaller molecular weight, which increases the stability of the complex and lowers cytotoxicity. Conjugating PEI to poly(cystaminebis(acrylamide)- diaminohexane) (poly(CBA-DAH)) via a disulfide bond further decreases toxicity and allows genetic material to be released easily.
Stage of Development
Lead Identified
Benefit
- Facilitates delivery of genetic materials such as pDNA, siRNA, and pshRNA.
- Improves transfection efficiency (up to 10x that of Lipofectamine).
- Reduces toxicity.
- Improves gene silencing efficiency.
- Enables additional in vivo applications through increased biocompatibility.
Publications
Nam, H.Y., Nam, K., Lee, M., Kim, S.W., Bull, D.A. (2012).
Dendrimer type bio-reducible polymer for efficient gene delivery.
Journal of Controlled Release. 160(3):592-600.
IP
Publication Number: US 2015-0057339 A1
Patent Title: High Molecular Weight Arginine-Grafted Bioreducible Polymers
Jurisdiction/Country: United States
Application Type: Non-Provisional
Contact Info
Aaron Duffy
(801) 585-1377
aaron.duffy@utah.edu