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Transient Gene Therapy for Neuroprotection

Description:

Safe, minimally-invasive, non-viral delivery of nucleic acid into the central nervous system (CNS) to protect cells from ischemic or traumatic injury

 

Inventor

Dr. James G. Hecker

 

Problem

Gene therapy has significant potential to advance clinical medicine. However, long-term gene expression delivered by viral modes may contribute to risks and duration of expression must be matched with the appropriate indication. Transient expression to address acute indications, such as neuroprotection after ischemic or traumatic injury, could significantly aid in recovery and prevent long-term effects.

 

Solution

Dr. Hecker has developed a non-viral cationic lipid-based delivery system to address transient gene therapy needs. The application is minimally invasive, through injection into the CNS, and is highly effective in transfection of both neuronal and non-neuronal cells. 

 

Advantages

Low immunogenicity

Easy preparation

No limitations on vector size

 

Stage of Development

In vivo proof of concept

 

Intellectual Property

UP Application (US20100249208 A1) – received notice of allowance 7/2015

 

Reference Media

Hecker et al. Molecular Therapy (2008) 16 11, 1857–1864

 

Desired partnerships

• License

 

Download PDF

 

Docket #  P3141 


Patent Information:
For Information, Contact:
Neal Lemon
Associate Director, PSOM Licensing Group
University of Pennsylvania
nlemon@upenn.edu
Inventors:
James Hecker
Keywords: