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Splice Modulating Oligonucleotides as a Breast Cancer Therapy

UCR researchers have designed novel splice modulating oligonucleotides (SMOs) that decrease expression of the long form of the prolactin receptor, thereby significantly inhibiting the metastatic spread of breast cancer to the lungs and liver. The SMO treatment also increased central death in the primary breast tumor. These SMOs may also target metastases produced by non-prolactin receptor-expressing primary tumors since all cancer stem cells examined so far are positive for the prolactin receptor. The researchers administered SMOs to two highly aggressive metastatic models of breast cancer, BT474 human xenografts, used for testing Herceptin, and a 4T1 syngeneic mouse model, which allows testing with an intact immune system.   Fig. A shows a reduction in the number of metastatic colonies upon treatment with the UCR SMOs.       Fig. B titled "Control" is a stain of the metastatic colonies without SMO treatment. Fig. B titled "PRLR SMO" is a stain of the colonies after 40 days treatment with the UCR SMOs.

Identification Of A Factor That Promotes Human Hematopoietic Stem Cell Self-Renewal

The Mikkola group at UCLA has discovered a novel regulator of hematopoietic stem cell self-renewal. The overexpression of this regulator increases the yield of ex vivo stem cell expansion and could thereby improve the efficiency of stem cell therapies. 

A Mouse Model of Human Papillomavirus (HPV) infection for Drug Discovery

UCSF researchers have generated and validated a K14-HPV16 transgenic mouse model, in which transgene expression produces neoplastic progression that fully resembles the gynecological and other epithelial dysplastic lesions induced by high risk HPVs. This model offers an invaluable tool for studying HPV infection and developing new drugs for HPV treatment.

Mesenchymal Stem Cell Derived Exosomes for Treating Peripheral Artery Disease

Researchers at the University of California, Davis have developed a method to isolate exosomes from mesenchymal stem cells that contain signaling molecules that induce angiogenesis. The isolated exosomes can be used for treating peripheral arterial disease.

A Micro/Nanobubble Oxygenated Solutions for Wound Healing and Tissue Preservation

Soft-tissue injuries and organ transplantation are common in modern combat scenarios. Organs and tissues harvested for transplantation need to be preserved during transport, which can be very difficult. Micro and nanobubbles (MNBs) offer a new technology that could supply oxygenation to such tissues prior to transplantation, thus affording better recovery and survival of patients. Described here is a novel device capable of producing MNB solutions that can be used to preserve viability and function of such organs/tissue. Additionally, these solutions may be used with negative pressure wound therapy to heal soft-tissue wounds.

Compositions and Methods using RNA Splicing Modulation to Selectively Impair Leukemic Cancer Stem Cells

The advancing age of the US population and increasing exposure to chemotherapy (for other malignancies) has resulted in increased rates of myelodysplastic syndrome (MDS). Following on the heels of MDS is progression to therapy-resistant acute myeloid leukemia (AML), which is predicted to rise significantly over the next few decades.  The heterogeneity of molecular abnormalities in therapy-resistant secondary acute myeloid leukemia (sAML) combined with a paucity of effective treatment options has resulted in high relapse-related mortality rates. In addition to approved therapies, many experimental agents also target epigenetic regulators of gene expression in clinical trials for sAML. However, most of these agents fail to improve patient survival, suggesting that epigenetic modifier therapies may reduce leukemic burden but may not effectively target a subpopulation of therapy-resistant leukemia stem cells that drive relapse. Hence, there is a critical need for developing clinical candidates with different modes of action. Recent studies implicate the spliceosome as a therapeutic vulnerability in solid tumors.

Deployable Applicators for Ultrasound Therapy

This invention is a modified catheter-based therapeutic ultrasound device with a novel combinatorial assembly of ultrasonic sources, deployable acoustic reflectors, and a fluid lens intended for the delivery of acoustic energy to target tissue. This assembly overcomes restrictions on dimensions required for minimally-invasive surgical introduction into a body cavity or lumen, and when deployed improves depth and range of treatment delivery for hyperthermia, thermal ablation, drug delivery, or sonotherapy.

Remotely-Activated Cell Therapy

The remote control of cellular activation in a controllable and reproducible fashion is a key tool for biological research, as well as for therapeutic uses. Cellular therapies are becoming well established within the medical community. However, the degree of cellular activation can be an unknown factor, and the risk of off-target effects remains. Cells may be delivered, but may not be therapeutically effective, or effective cells may elicit activity in an undesired location. The delivery of a cell therapy where a known quantity of cell activation occurs at a specific, selected site may therefore be advantageous. UC San Diego researchers have recently developed the methods and materials for remote control of cellular activation, to dynamically manipulate molecular events for therapeutic effect.

Safe And Targeted Electric Stimulation Of The Human Cranial Nerves

Neuromodulation (electrical stimulation of the nervous system) is used in cochlear and retinal implants, or deep brain stimulation devices to treat various neurological disorders (i.e. depression, Parkinson’s Disease). However, such approaches tend to be invasive and expensive. Researchers at UCI have developed a novel approach and device to stimulate the cranial nerves that is targeted, safe, and minimally-invasive for the treatment of diseases or the activation of senses.

Repurposing Dabuzalgron to Prevent and Treat Cardiomyopathy and Heart Failure

Using an alpha-1A-adrengic receptor agonist, dabuzalgron, as a therapeutic treatment for cardiomyopathy and heart failure.

Novel method for detection of O-Sulfonation sites on post-translationally modified proteins

Sulfonation of proteins and carbohydrates plays an important role in signaling, transport, and metabolism in the body. The degree to which a molecule is modified and at what positions dictates how that structure interacts within the body. UCI researchers have developed novel methods of detecting and mapping serine and threonine sulfonation of peptides and proteins.


The invention is a method for instantaneous and efficient extraction of radioactive isotopes with high specific activity, during continuous production at research reactors. The proposed method allows advantageous production of radioisotopes for various applications, including nuclear medicine uses (diagnostics, imaging, cancer treatments). In addition, the invention has the potential for applications related to isotopes used in thermoelectric generators (i.e. 238Pu) that power both medical devices, such as cardiac pacemakers, and deep space missions.

Reduced IP3 Signaling As A Diagnostic Tool For Autism Spectrum Disorders

The diagnosis of Autism Spectrum Disorder (ASD), and thus the development of therapies, is very challenging due to the lack of objective criteria and biomarkers. It is, however, a disease with a strong genetic component, and recent data has implicated new genes in the disease. Researchers at UC Irvine have developed a method to more reliably diagnose ASD with a laboratory test.

Hydrogel for Improved Burn Wound Healing

Background: The US spends $25B alone on wound treatment of burns. The wound treatment market is projected to grow to $18.3B in just 4 years. Current treatments have been unsuccessful in fostering proper wound healing safe from infections. Not only do they have low efficacy, but they are very expensive to produce. The most commonly used wound dressing is a hydrogel. Hydrogels reduce pain and healing time, promotes cell proliferation and collagen deposition, and is the most appropriate for burn wounds.  Brief Description: UCR researchers have developed a novel formulation that can be embedded into a hydrogel to significantly improve wound healing. Through successful conjugation of a peptide and polymer, stability and longevity have been enhanced. Their formulation induced a 3-fold increase in density of newly formed microvessels, greatly improving tissue quality.

Selective Inhibition of Activated ErbB Tyrosine Kinases

This invention identifies a novel class of ErbB targeting small molecules.

C3d-binding Biomarkers for Detection of Complement-mediated Inflammation

Background: The complement immune system is implicated in many acute and chronic inflammatory conditions and autoimmune diseases, including neurological (Alzheimer’s and multiple sclerosis), renal (lupus nephritis and glomerulonephritis), ocular (age-related macular degeneration), and systemic (lupus and rheumatoid arthritis). The complement protein C3d resides covalently attached in inflamed tissues, and it is an excellent biomarker target for complement-mediated inflammation, even at early disease stages prior to clinical manifestations.  Brief Description: UCR researchers have discovered several small chemical compounds with intrinsic fluorescence properties that bind to complement C3d. These compounds can serve as molecular biomarkers for the detection of complement activation using fluorescence imaging. The compounds can be developed to become noninvasive in vivo diagnostics of complement-mediated inflammatory and autoimmune diseases, for spatiotemporal monitoring of disease progression, and for delivering therapeutics to sites of inflammation.

Screening Platform for Anti-influenza Drugs

In addition to their protective functions as lubricants and physical and immunologic barriers, recent work has demonstrated that mucins also provide protection from influenza virus infection by presenting decoy ligands for the virus. In airway passages, target glycans on airway cells are covered with a thick layer of mucus containing dense array of glycosylated proteins (mucins). However, most screens are either overly simplified (and therefore not representative of the natural mucin barrier) or not translatable to a high-throughput platform. Hence, the development of a useful high throughput drug screening method has been hindered by the inability to streamline the process of replicating the complex environment that airborne invaders naturally confront.

Technology For Sustaining Pluripotency And Improved Growth Of Stem Cells In Culture

Background: Pluripotent stem cells (PSC) have tremendous potential in regenerative medicine, cell therapy, and drug/toxicant screening, and can increase our understanding of the pathogenesis and treatment of disease. The stem cell industry has accelerated annual growth projections of 20% by 2020. Overall, there is growing demand for culture media that can support rapid growth, survival, and sustain pluripotency of stem cells. Brief Description: UCR researchers have developed a novel, non-toxic biological compound that can be added to any culture medium to prevent unwanted differentiation. Compared to the standard commercial media currently used in PSC laboratories, this compound produces major improvements in cell quality, cell growth and maintenance of pluripotency during repeated passaging. Additionally, regulatory groups categorize stem cell therapy as an orphan drug, thereby allowing accelerated approval.

Synthetic Lethal Targeting Of Rho-Associated Kinase

Clear Cell Renal Cell Carcinoma (CC-RCC) is the most common form of kidney cancer in adults. Researchers at UCI have identified novel chemical compounds and methods for treating CC-RCC by targeting and killing cancerous cells without affecting healthy cells.

Inhibitors Of Ires-Mediated Protein Synthesis

The Gera group at UCLA has discovered a novel analog of a known compound with significant anti-glioblastoma potential when used in combination with mTOR inhibitors.

Methods For Promoting Oligodendrocyte Regeneration And Remyelination

Researchers at the University of California Davis have demonstrated that immature astrocytes generated from human pluripotent stems cells, promote oligodendrocyte lineage progression via TIMP-1 secretion.

A Novel Therapeutic Against HIV Using Human T Cell Immunoglobulin Mucin (TIM-3) Ligands to Modulate Immune Response

Blocking human T cell immunoglobulin and mucin domain-containing molecule 3 (TIM-3) signaling can restore functionality to defective T cells in HIV-1 infected patients. Additionally, measuring TIM-3 provides clinicians with a novel way of evaluating, staging, and monitoring the progression of HIV infections.

miRNA Therapeutic for Huntington's Disease

Huntington's disease (HD) is a neurodegenerative genetic disorder caused by the mutation of a CAG repeat within the HD gene that causes the accumulation of mHTT species. The biochemical pathways involved in HD are complex and not yet fully uncovered. As a consequence, there are currently no successful disease-modifying therapies for HD. The identification of cellular targets that impact the disease's onset and progression is critical for the development of new treatments. Researchers at UCI and University of Iowa have identified a new target and developed an miRNA therapeutic that reduces accumulation of mHTT in a mouse model of HD.

Synthesis of Lipobactins and Teixobactin Analogues – New Antimicrobial Compositions against Gram-Positive Bacteria

With the discovery of penicillin in the 1940’s, many scientists proclaimed the defeat of infectious diseases which had plagued mankind. However, the remarkable healing power of antibiotics unfortunately invited widespread and indiscriminate use of antibiotics. This misuse and overuse of antibiotics has led to the dramatic rise in antibiotic resistant bacterial strains and increased healthcare costs.

Method to Enrich for Cells Transduced with Chimeric Antigen Receptors

Researchers at UCLA have developed a method to expand chimeric antigen receptor-transduced T cells for use in immunotherapies.

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