Vectors for the Treatment of Sickle Cell Disease and Beta Thalassemia

This technology includes lentivirus vectors to be used to treat sickle cell disease and beta thalassemia. (i) Lin28A or Lin28B vectors designed for erythroid-specific expression using EKLF1, SPTA1, or similar erythroid-specific regulatory elements will be used to transduce hematopoietic stem cells isolated from humans with sickle cell disease or beta-thalassemia syndromes.

Transgenic Mice with Conditionally Activated Islet Beta Cell M3 Muscarinic Acetylcholine Receptor for Improving Glucose Tolerance in High-fat Diet Obese Insulin-resistant Mice

This technology includes transgenic mice in which designer rat M3 muscarinic receptor mutants were expressed only in islet 13-cells (directed by rat insulin promoter II), were unable to bind acetylcholine (the endogenous ligand) but could be selectively activated by an otherwise pharmacologically inert compound (clozapine-N-oxide (CNO)). The R-q receptor contained a Y148C point mutation, which enabled CNO to selectively activate G proteins of the Gq/11 family. The R-5 receptor contained an A238G mutation, which enabled CNO to selectively activate G proteins of the G5 family.

Identification and Characterization of the Wild Mouse Gut Microbiome as the Optimal Standard for Laboratory Mice

This technology includes identification of the wild mouse microbiome as a method to increase resistance to lethal viral infection. We establish that the gut microbiome of barrier-raised C57BL/6 mice is dysbiotic compared to that of their outbred, wild-living progenitors, Mus musculus domesticus. We find that the multigenerational offspring of pregnant germfree C57BL/6 mice reconstituted with the gut microbiome of wild mice exhibit a less inflammatory response and increased survival following influenza A virus infection.

In-vivo System to Interrogate the Functions of Mucous Membranes and Identify Mucin/Glycan Mimetics and JAK/STAT Inhibitors for the Treatment of Diseases of the Oral Cavity and Digestive Tract

This technology includes a Drosophila mutant strain that can be used as an in vivo model for diseases of the oral cavity and digestive tract (Sjogren's syndrome, colitis, colon cancer, inflammatory bowel disease), where the mucous membrane is disrupted or non-functional. This mutant lacks a mucous membrane and displays epithelial cell damage, uncontrolled cell proliferation and the up-regulation of conserved signaling pathways (JAK/STAT).

KCNN4 Knockout Mice for Mechanistic Research

This technology includes a transgenic allele for a mouse knockout model for the KCNN4 gene. Secretion of fluids from these salivary glands requires the coordination of multiple water and ion channel proteins. Notably, the majority of these channels have been shown to be up-regulated by increased calcium concentrations. The relevant calcium-activated potassium channels are split into the small, intermediate, and large conductance channels (called the SK, IK, and BK channels). The KCNN4 gene plays a part in the IK and BK channels.

Application of AAV44.9 Vector in Gene Therapy for the Inner Ear

This technology includes a novel AAV isolate (AAV44.9) to be used as gene therapy for the inner ear for the treatment of deafness. The ability of AAV vectors to transduce dividing and non-dividing cells, establish long-term transgene expression, and the lack of pathogenicity has made them attractive for use in gene therapy applications. Vectors based on new AAV isolates may have different host range and different immunological properties, thus allowing for more efficient transduction in certain cell types.

Locally Delivered Alkaline Phosphatase for Treatment of Periodontal Disease

This technology includes a product for local delivery of alkaline phosphatase for the treatment of periodontal disease. Our laboratory has discovered that factors regulating phosphate metabolism and specifically the appropriate balance between phosphate (Pi) and pyrophosphate (PPi) at local sites are needed for formation (development), maintenance and regeneration of the tooth root surface (cementum), periodontal ligament (PDL) and surrounding alveolar bone, i.e., the periodontal apparatus.

High-Resolution and Artifact-Free Measurement and Visualization of Tissue Strain by Processing MRI Using a Deep Learning Approach

This technology includes a system for automatic artifact-free measurement and visualization of tissue strain by MRI at native resolution. The investigation of regional soft tissue mechanical strain can serve as a unique indicator for different related disorders. For example, measurement of myocardial tissue during contraction can help calculate, track, and assess cardiac stress. Currently, methods such as tagging MRI (tMRI) are used for imaging soft tissue deformation. Despite being well validated, methods such as tMRI suffer from low spatial and temporal resolution.

Multiplexing Homocysteine in Primary Newborn Screening Assays Using Maleimides as Select Derivatization Agents

Homocystinuria (HCU), a group of inherited disorders, causes symptoms ranging from failure to thrive and developmental delays in infants or young children to abnormal blood clots with onset in adults.1 Approximately 1 in 200,000 to 335,000 people have HCU globally.2

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.
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