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This technology includes a novel family of adenosine kinase (AdK) inhibitors, including pharmaceutical compositions containing the adenosine kinase inhibitors, and their use for preventing epilepsy and its progression in patients. Endogenous adenosine (i.e., naturally occurring adenosine) acts on G protein-coupled receptors (adenosine receptors, ARs) in the central nervous system to suppress seizures and pain, and to blunt the effects of ischemia (a restriction in blood supply to tissues).

This technology includes a conventional knockout mice: beta- 1,4-N-acetylgalactosaminyl transferase 1 (GM2 Synthase) KO; B4galntltm1Rlp for the study of glycosphingolipid storage disorders. The glycosphingolipid (GSL) storage diseases are caused by genetic disruption in the lysosomal degradation pathway of GSLs, and include Tay-Sachs disease, Sandhoff's disease, Gaucher's disease, Fabry's disease, Krabbe's disease, and several others. In most of these diseases, GSLs accumulate to massive levels in cells, particularly in neurons, causing neurodegeneration and a shortened life span.

This technology includes the use of selective antagonist for the P2Y14 receptor for the treatment and prevention of neuropathic pain. Neuropathic pain conditions arising from injuries to the nervous system due to trauma, disease or neurotoxins are exceedingly difficult to treat. Clinicians and patients are often left to manage neuropathic pain with opioids, but these approaches are limited by the eventual loss in opioid efficacy with developing tolerance, the occurrence of severe adverse side effects and the strong potential for their abuse.

This technology includes A1AR-selective agonists which are full or partial agonists of the A1AR and are being considered for treatment of various conditions: seizures, stroke, diabetes, pain, cardio-protection and arrhythmias. A1AR agonists are highly neuroprotective in ischemic and epileptic models. A1AR agonists are also being explored for antidepressant, antianxiety, and other neuropsychiatric effects, due to their presynaptic action to decrease the release of excitatory amino acids in the brain.

This technology includes a cell line to be used for the study of anti-psychotic therapies and potentially Parkinson’s disease. Activation of D1 dopamine receptors plays a critical role in many fundamental CNS processes. M4 mAChRs are coexpressed with D1 dopamine receptors in a specific subset of striatal medium spiny neurons that contain GABA as the major neurotransmitter. The present study used Cre/LoxP technology to generate mutant mice that lack M4-¬-AChRs only in D1 dopamine receptor-¬-expressing cells to investigate the physiological relevance of mAChRs in this neuronal subpopulation.

This technology includes (N)-methanocarba derivatives that are selective agonists of the A3 receptor to be used for the treatment of chronic neuropathic pain. 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 new nucleoside inhibitors containing rigid rings that provide high potency for use as antiepileptic drugs. Adenosine kinase (AdK) inhibitors raise the level of endogenous adenosine, particularly in disease states, and are of interest for the potential treatment of seizures and neurodegenerative and inflammatory conditions.

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.

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.

Many experimental therapies will rely on the local parenchymal delivery of macromolecules or nucleic acids for their success. However, the volume of distribution of many of these potential therapeutic agents is restricted by their interactions with the extracellular matrix and cellular receptors.