AAV based viral vectors that express either AAV-NG2Ab or AAV-NT3 have been shown to provide improved recovery of motor function in models. Background: Spinal Cord Injury (SCI) affects nearly 270,000 Americans and is a common cause of permanent disability and death in children and adults. Despite being a significant burden to the patients, society and healthcare industry, SCI currently has no treatment. It is known that, upon SCI, macrophages/ microglial cells rush to the site and contribute in development of inflammatory response at the injury epicenter. Therefore manipulating these first responders to deliver desirable proteins such as
neutralizing antibodies to inhibitory proteins, or
to the site of injury represents an excellent therapeutic strategy. Technology Overview: Researchers at Stony Brook University have developed AAV based viral vectors that express either NG2-neutralizing antibody (AAV-NG2Ab) or Neurotrophins (AAV-NT3) and have been shown to provide improved recovery of motor function in relevant animal models. The vectors have high tropism for both neuronal and glial cells and carries a promising potential as gene therapy vector for SCI and other neurodegenerative injuries.
Petrosyan et al., J Nerrosci, 33(9):4032-43, 2013
Petrosyan et al., Gene Therapy, 21:991-1000, 2014 Advantages: Significant recovery in locomotor function in relevant animal models
High transduction efficacy in glial and neuronal cells
No adverse inflammatory response following vector administration Applications: Brain injury
Spinal cord injury
Neurodegenerative injuries Intellectual Property Summary: Patented,Patent application submitted Stage of Development: Issued US Patent # 9,623,107 (Issue date 04/08/2017) Pending continuation application # 15/448,94 Licensing Potential: Licensing,Development partner,Commercial partner Licensing Status: Available for License. Stony Brook University seek to develop and commercialize, by an exclusive or non-exclusive license agreement and/or sponsored research, with a company active in the area. #8563 Additional Information: spinal,spinal cord,spinal cord injury,macrophage,microglial,antibody,therapeutic,vector,motor function,neuronal,glial cell,therapies,gene therapy vector,neurodegenerative,transduction,neuronal cell,brain injury,aav gene therapy,aav vector,aav,adeno associated viral vector,adeno-associated,adeno-associated virus,adeno-associated virus vector,spine,vector delivery,SCI,neuronal cells,gene therapy,gene therapy development,neural,neural degeneration,neurodegeneration,neurodegenerative gene therapy,neurodegenerative treatment https://stonybrook.technologypublisher.com/files/sites/y6teltcwrcmo1jeyycx0_aav2.png Please note, header image is purely illustrative. Source: Jazzlw, Wikimedia Commons, CC BY-SA 4.0.