Potency Assay for Membrane Transporter Protein-based Drugs Acting on Antioxidant, Redox, and Apoptosis Response Pathways

Description of Technology:

This technology includes a method of analyzing the potency of membrane transporter protein-based drugs acting on intracellular antioxidant and redox response pathways (and associated apoptosis pathways), wherein the drug delivery and activity is lipid associated. The present invention is a cell-based bioassay for measuring the bioactivity of drug substance and formulated drug product by determining the drug's dose-dependent inhibitory effects on 4 hydroxynonenal (4-HNE)-induced antioxidant response element (ARE) activity.

Mouse Embryo Culture Chamber and Imaging System and Methods of Use

Description of Technology:

The culture of mouse embryos ex utero and continuous monitoring and imaging of embryos as they develop have applications in drug testing, genetic studies, and basic research on embryonic development. However, the embryo culture systems currently available for post-implantation embryos include rolling bottle culture systems, which do not permit imaging of the developing embryos and do not support the long-term survival and development of embryos ex utero.

Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies

Description of Technology:

Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype and expression of phototransduction genes in an in vitro model of CRX-LCA.