A Layer-by-Layer Approach to Co-deliver DNA and siRNA Via AuNPs

Case ID:
C12219
Disclosure Date:
10/22/2012
Description:

C12219: Gold Nanoparticles for Gene Therapy

Novelty:

This technology is a bioreducible/biodegradable polymer coated gold nanoparticle (AuNP) for the co-delivery of siRNA and DNA for targeted gene therapy.

Value Proposition:

There is a current unmet medical need for methods to safely and efficaciously deliver nucleic acids to cells and tissues for gene therapy. Approaches involving the use of viral vectors or polymeric vectors are limited by either their immunogenicity or lack of efficacy. The invention addresses these concerns and provides efficacious delivery of nucleic acids for targeted gene therapy. Advantages of this technology include:

• Enhanced transfection of AuNP into target cells
• Simultaneous delivery of siRNA and DNA which can be used to target various diseases
• Gene expression and knockdown can be kinetically controlled based on arrangement of polymers on AuNP
• Rapid and efficient release of nucleic acids into the cellular compartment

Technical Details:

Johns Hopkins researchers have developed methods to synthesize AuNPs complexed with 2 unique cationic polymers and 2 anionic nucleic acids. It involves the layer-by-layer addition of cationic polymers, with different degradable properties, and anionic nucleic acids to the AuNPs. The 2 nucleic acids can be siRNA and DNA. The 2 polymers are a hydrolytically degradable ester and a disulfide-reducible amine. The ester polymer enhances transfection efficiency by its buffering properties (“proton sponge effect”) which enables the AuNP to escape from the endosome, thus preventing degradation of nucleic acid cargo. The bioreducible amine polymer enables rapid release of the nucleic acid cargo into the cytoplasmic environment where it can exert its effect. The invention provides opportunities for targeting various diseases involving genetic factors.

Looking for Partners:

To develop & commercialize the technology as a research tool for targeted gene delivery.

Stage of Development:

Discovery

Data Availability:

In vitro data in Human Glioblastoma Multiforme cell lines

Publications/Associated Cases:

“Not at this time”

Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
A Layer-by-Layer Approach to Co-deliver DNA and siRNA Via AuNPs PCT: Patent Cooperation Treaty United States 14/438,348 4/24/2015     Pending
Inventors:
Category(s):
For Information, Contact:
Sonriza Ford
sford23@jhu.edu
410-614-0300
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