Treatment of Immune-mediated Brain Swelling with Combined Anti-LFA1/VLA4 Therapy

This technology includes a therapeutic approach to prevent secondary edema after cerebrovascular hemorrhage. Using an animal model, we found that edema is triggered by massive extravasation of myelomonocytic cells from the blood into the brain in response to hemorrhaging vessels. Administration of anti-LFA1 and anti-VLA4 antibodies resulted in an inhibition of extravasation of the myelomonocytic cells. This single dose treatment prevented secondary edema and markedly improved functional outcomes if administered within the first six hours following cerebrovascular hemorrhage.

Targeting the 5’UTR of Survival Motor Neuron 2 (SMN2) with Antisense Oligonucleotides to Increase Expression for the Treatment of Spinal Muscular Atrophy

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.

Preparation of Substituted Diarylpropanamides as RORgt Antagonists for the Treatment of Th17-related Autoimmune Diseases

This technology includes a series of diphenylpropanamides as potent and selective RORgt inhibitors for the treatment of Th17-related autoimmune diseases. The retinoic acid-related orphan receptor RORgt plays an important role in the differentiation of thymocytes, lymphoid tissue inducer cells, and inflammatory T helper-expressing interleukin 17a (Th17) cells. Small molecule RORgt inhibitors may provide means to regulate Th17 mediated immune response. The novel molecules have potential to treat Th17-related autoimmune diseases.

Discovery of imidazo[1,2-b]pyridazines with Anticancer Properties

This technology includes a series of imidazo[1,2-b]pyridazines that display potent inhibition of FLT3, as well as potent binding and activity against FLT3 tyrosine kinase domain and gatekeeper mutations. This chemotype exhibits superior anti-leukemic activity against the common clinically-relevant FLT3-mutant acute myeloid leukemia (AML) in vitro and in vivo. Tyrosine kinase domain mutations are a common cause of acquired resistance to FLT3 inhibitors used to treat FLT3-mutant AML.

Gene Therapy for Cobalamin C Deficiency (cblC) with Viable Mouse Models

Cobalamin C deficiency (cblC) is the most common inborn error of intracellular cobalamin metabolism and is caused by mutations in MMACHC, a gene responsible for processing and trafficking dependent enzymes: intracellular cobalamin, resulting in elevated methylmalonic acid and homocysteine and methionine deficiency. Disease manifestations include growth failure, anemia, cardial defects and progressive blindness.

Handwipe Disclosing Method for Detecting the Presence of Lead

Lead (Pb) exposure can cause serious health concerns including abdominal pain, headaches, loss of appetite, memory loss, weakness, and other symptoms. Lead residues on human skin, especially on the hands of workers can be a significant health risk since such residues may be ingested during normal activities (e.g. eating, drinking, and smoking). A key component to reducing lead exposure is being able to identify areas of lead contamination.

Wipes and Methods for Removal of Lead and Other Metal Contamination from Surfaces

Exposure to lead (Pb) has long posed serious health risks. Ingestion of lead from skin exposure can adversely impact every organ in the body; the kidneys, blood, nervous, and reproductive systems are most affected. Washing skin with soap and water is not sufficient to remove lead residues. To prevent adverse impacts from Pb exposure, exposed individuals need cleaning methods that will effectively remove Pb ions from the skin to less than the limit of identification (i.e., 10 µg or less).

Methods of Predicting Patient Treatment Response and Resistance via Single-Cell Transcriptomics of Their Tumors

Tailoring the best treatments to cancer patients remains a highly important endeavor in the oncology field. However, personalized treatment courses are challenging to determine, and technologies or methods that can successfully be employed for precision oncology are lacking.

Molecular Nanotags for Detection of Single Molecules

Biological nanoparticles, like extracellular vesicles (EVs), possess unique biological characteristics making them attractive therapeutic agents, targets, or disease biomarkers. However, their use is hindered by the lack of tools available to accurately detect, sort, and analyze. Flow cytometers are used to sort and study individual cells. But, they are unable to detect and sort nanomaterials smaller than 200 nanometers with single epitope sensitivity.

Exo-Clean Technology for Purifying Extracellular Vesicle Preparations from Contaminants

Extracellular Vesicles (EVs), including exosomes and microvesicles, are nanometer-sized membranous vesicles that can carry different types of cargos, such as proteins, nucleic acids and metabolites. EVs are produced and released by most cell types. They act as biological mediators for intercellular communication via delivery of their cargos. This unique ability spurred translational research interest for targeted delivery of therapeutic molecules to treat a wide range of diseases. EVs also contain interesting information of their specific cellular origin.

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