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This technology includes six human monoclonal antibodies (mAbs) that target tumor antigens derived from the CT-RCC HERV-E (human endogenous retrovirus type E) to generate Chimeric Antigen Receptor (CAR) T cells to treat patients with advanced clear cell renal cell carcinoma (ccRCC). These mAbs were identified from Adagene Inc’s human antibody phage library, and data show that majority of these mAbs only bind to CT-RCC HERV-E+ ccRCC cells, which express TM but not CT-RCC HERV-E non-expressing ccRCC cells nor non-RCC cells.

This technology includes a transgenic reporter mouse that expresses a fluorescent protein called mt-Keima, to be used to detect and quantify in vivo mitophagy. This fluorescent protein was originally described by a group in Japan and shown to be able to measure both the general process of autophagy and mitophagy. We extended these results by creating a living animal so that we could get a measurement for in vivo mitophagy. Our results demonstrate that our mt-Keima mouse allows for a straightforward and practical way to quantify mitophagy in vivo.

This technology includes a generated polyclonal antibody in rabbit that detects the mitochondrial uniporter (MCU) protein. This antibody was created by immunizing rabbits with a synthesized sequence of the MCU protein and can be used to identify and quantify MCU protein in various tissues. The polyclonal nature of the antibody ensures it recognizes multiple epitopes on the MCU, enhancing detection reliability. This technology is crucial for understanding MCU's role in mitochondrial function and mammalian physiology.

This technology includes antibodies for TMC1 protein as a treatment for hearing loss. TMC1 is one of the common genes causing hereditary hearing loss. Our laboratory used synthetic peptides corresponding to the TMC1 protein to immunize rabbits. The resulting antisera were shown to bind to TMC1 protein expressed in heterologous expression systems. TMC1 protein is required for the transduction of sound into electrical impulses in inner ear sensory cells.

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.

This technology includes rhesus macaque induced pluripotent stem cells (iPSCs) lines from multiple animals and various types of cells to establish this pre-clinical model. iPSCs are a type of pluripotent stem cell that can be generated from adult somatic cells. The iPSC technology holds great potential for regenerative medicine. Before clinical application, it is critical to evaluate safety and efficacy in a clinically-relevant animal model. We propose that non-human primate models are particularly relevant to test iPSC-based cell therapies.

Researchers at the National Cancer Institute (NCI) have developed orthotopic allograft models for pancreatic cancer that utilize low passage primary pancreatic adenocarcinoma cells or tumor fragments implanted into the cancer-free pancreata of recipient syngeneic immunocompetent mice. Tumor development in these models is more synchronized, latency is substantially shortened, and tumors develop only in one location, as pre-determined by the choice of a site for cells/tumor fragment implantation.

Current therapies for glioblastoma multiforme (GBM), the highest grade malignant brain tumor, are mostly ineffective, and better preclinical model systems are needed to increase the successful translation of drug discovery efforts into the clinic. Scientists at the National Cancer Institute (NCI) have developed and characterized an orthotopic genetically engineered mouse (GEM)-derived model of GBM that closely recapitulates various human GBM subtypes and is useful for preclinical evaluation of candidate therapeutics.

This technology includes an alpha-galactosidase-A knockout mouse model that can be used to study Fabry disease, an X-linked lysosomal storage disorder. Alpha-galactosidase-A is a crucial enzyme responsible for the breakdown of glycolipids, particularly globotriaosylceramide (Gb3), within lysosomes. In Fabry disease, a rare and inherited lysosomal storage disorder, mutations in the GLA gene lead to deficient or non-functional alpha-galactosidase-A enzyme activity.