Generation of AAVS1 and C13 “Safe Harbor” Transcription Activator-life Effector Nucleases (TALENs) for Drug Screening or Gene Therapy Development

This technology includes AAVS1 and C13 “safe harbor” transcription activator-life effector nucleases (TALENs) for drug screening or gene therapy applications. TALENs are engineered sequence-specific DNA endonucleases that can significantly enhance genome-editing efficiency by >100-1000 folds. “Safe harbor” such as AAVS1 safe harbor and C13 safe harbor is genome locus that allows robust and persistent transgene expression with no or minimal interference of endogenous gene expression and cell properties.

Resolution Doubling with Digital Confocal Microscopy

This technology includes a microscopy method that reduces the speed penalty at least 1000-fold, while retaining resolution improvement. A Digital mirror device (DMD) or sweptfield confocal unit is used to create hundreds to thousands of excitation foci that are imaged to a sample mounted in a conventional microscope and record the resulting emissions on an array detector. Detection of each confocal spot is done in our proprietary software, as is the processing and deconvolution that is used for a 2x resolution enhancement.

Instant Total Internal Reflection Fluorescence/Structured Illumination Microscopy (instant TIRF/SIM)

This technology includes a method which enables high-speed, super-resolution microscopy at a very high signal-to-noise ratio (SNR), for biological applications within ~200 nm (the evanescent wave decay length) of a coverslip surface. Instant TIRF/SIM may be implemented simply by modifying and adding to the excitation optics that are already present within a conventional instant SIM design. We enforce TIRF excitation by removing all wave vectors that propagate into the objective lens at sub-critical angles.

Treatment of the beta-globinopathies through inhibition of RIOK3 activity

Disorders of adult beta-globin synthesis, which include sickle cell disease (SCD) and beta-thalassemia, are the most common monogenic disorders in the world. While the curative potential of bone marrow transplantation has been demonstrated, this approach is limited to a small fraction of affected patients due to the requirement for an HLA-matched donor, the highly specialized approach that requires critical infrastructure, and the high cost.

Programmable and Modular Nucleic Acid Nanoassemblies-based (NAN) Platforms to Regulate Mechanosensitive Activation of T-cells

This technology includes mechanobiological nucleic acid nanoassemblies-based platforms with dynamically controlled efficiency of T-cell activation. T-cells are the central players in adaptive immune response led by a T-cell receptor (TCR) centric machinery. Current T-cell activation strategy (e.g., micron-scale beads) focuses on 2D TCR-agonist biomimetic surfaces and biomimetic 2D immune synapses with planar traction, which requires non-physiological hyper-stimulatory cytokines levels (e.g., IL-2), and thus, is incompatible with clinical applications.

Minibody for Conditioning prior to Hematopoietic Stem Cell and Progenitor Cell Transplantation

Patient conditioning is a critical initial step in hematopoietic stem and progenitor cell (HSPC) transplantation procedures to enable marrow engraftment of infused cells. Conditioning regimens have traditionally been achieved by delivering cytotoxic doses of chemotherapeutic agents and radiation. However, these regimens are associated with significant morbidity and mortality, and cannot be used safely in elderly or subjects with comorbidities.

Immunoassay-derived Protein Biomarkers of Atherosclerotic Cardiovascular Disease Risk

This technology includes a combination of 6 protein biomarkers and clinical risk factors to be used as an In Vitro Diagnostic Multivariate Index Assay (IVDMIA) that can improve the identification of individuals at high risk for atherosclerotic cardiovascular disease (ASCVD). Incorporation of novel protein biomarkers of ASCVD risk into risk assessment algorithms may improve their ability to identify individuals at high risk for ASCVD.

Mass Spectrometry Derived Protein Biomarkers of Atherosclerotic Cardiovascular Disease Risk

This technology includes a combination of protein biomarkers and clinical risk factors to be used as an In Vitro Diagnostic Multivariate Index Assay (IVDMIA) that can improve the identification of individuals at high risk for atherosclerotic cardiovascular disease (ASCVD) and myocardial infarction (MI). Incorporation of novel protein biomarkers of ASCVD risk into risk assessment algorithms may improve their ability to identify individuals at high risk for ASCVD.

Compositions and Methods for Reducing Serum Triglycerides

This technology includes a vaccine for lowering plasma triglycerides by inducing the formation of autoantibodies against either ANGPTL3 or ANGPTL4, which are inhibitors of Lipoprotein Lipase. This was done by conjugating synthetic peptides based on ANGPTL3 or ANGPTL4 to virus- like particles (VLPS). Injection of the vaccine in animal models was shown to induce the autoantibody against the target and to lower plasma triglycerides.