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This technology includes the design and use of several nanobodies that bind to the SARS-CoV2 spike protein receptor binding domain and block spike protein interaction with the angiotensin converting enzyme 2 (ACE2) receptor. Nanobodies are 12-15 kDa single-domain antibody fragments that are more stable and easier to produce in large quantities compared to conventional antibodies. SARS-CoV2 is the virus responsible for the COVID19 pandemic. The SARS-CoV2 spike protein is responsible for viral entry into human cells via interaction with ACE2 on the cell surface.

The invention is a novel longer-lived mouse model for Gaucher disease. Gaucher disease is a genetic disorder that results from deficiencies in the enzyme glucocerebrosidase (GBA). The use of animal models to study how the disease progresses has been invaluable in research of this disorder. However, existing mouse models have been limited due to early mortality because the GBA enzyme plays an important role in lysosomal storage.

This technology includes the identification and use of antisense oligonuclecotides (ASOs) complimentary to the 5’UTR of SMN2 (Survival of motor neuron 2) for the treatment of spinal muscular atrophy (SMA). SMA is an autosomal-recessive motor neuron disease caused by the loss of both copies of the SMN1 gene. Copies of the similar gene SMN2 decrease the severity of this disease in a dose-dependent manner. Thus, increasing expression levels of the SMN2 transcript can be used to treat SMA.

This technology includes a sensitive and specific method to rapidly detect antibodies in biofluids. This assay has been used for the detection of antibodies in blood, urine, and saliva. Until now, no one has used LIPS to detect clinically relevant antibodies to SARS-CoV-2 Nucleocapsid (N) or Spike (S) in saliva. Briefly, LIPS employs recombinantly synthesized target proteins or peptides (e.g., S and N proteins) tagged with light-emitting proteins as targets to be captured by host produced immunoglobulins. These immunoglobulins can be captured by protein A/G beads and immobilized.

This technology includes microRNAs for use in cell lines for protein production and potentially future treatments of cancer or diseases related to metabolism. Mmu-miR-466h was identified as a major apoptotic regulator in suspension adapted Chinese Hamster Ovary cells. Mmu-miR-466h was found to have the pro-apoptotic activity by targeting some anti-apoptotic genes for degradation during the exposure of CHO-S cells to the nutrients depleted media.

This technology includes a compound for use as a selective agonist of the A1 adenosine receptor (AR) for therapeutic hypothermia and other conditions. We have examined various synthesized nucleosides in a model of mouse hypothermia, in conjunction with AR knockout mice, to characterize the biological profiles. In trying to identify novel highly selective A1AR agonists that have superior in vivo activities, we have adapted a means of rigidifying the ribose moiety of adenosine in the form of a bicyclic (N)-methanocarba ring.

This technology includes a method using ionophores to reduce sickling in patients with sickle cell disease. Sickle cell disease is caused by polymerization of a hemoglobin mutant, and the only approved treatment acts by replacing sickle hemoglobin with fetal hemoglobin, thereby increasing the delay time prior to polymerization. This drug is only partially successful because it does not induce fetal hemoglobin synthesis in all cells.

This technology includes compounds that are selective agonists of the A3 receptor for the treatment of various disorders such as cancer and autoinflammatory diseases. Structurally, these compounds extend the class of (N)-methanocarba derivatives that are selective agonists of the A3 receptor.

This technology includes a class of A3AR-selective agonists to be used therapeutically to treat a variety of conditions, including chronic pain, cancer, and inflammatory diseases. This class of compounds produced full agonists of the human A3AR of nanomolar affinity that were consistently highly selective (>1000-fold vs. A1AR and A2AAR). The selectivity at mouse A3 receptors is smaller, but the compounds are still effective in vivo in reducing or preventing development of neuropathic pain.

This technology includes three new chemical entities discovered for antibacterial and antifungal activities. The compounds are novel tetramic acid containing polyketides obtained from two different Amycolatopsis strains. Their planar structures and relative stereochemistry were elucidated by 1D and 2D NMR methods, including 1H-1H and 13C-13C COSY, TOCSY, HSQC, HMBC and ROESY. Whole genome sequencing of these two strains revealed a 158 kb biosynthetic gene cluster (BGC) containing a 23-module, mixed NRPS-PKS pathway responsible for their biosynthesis.