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Identification And Development Of Dual nSMase2-AChE Inhibitors For Neurodegenerative Disorders

UCLA researchers in the Department of Neurology, and the Department of Chemistry & Biochemistry have developed small molecule inhibitors of both the neutral sphingomyelinase 2 (nSMase2) and acetylcholinesterase (AChE) as novel therapeutics for neurodegenerative disorders caused by protein aggregation.

Carborane-Based Histone Deacetylase (HDAC) Inhibitors

UCLA researchers from the Department of Chemistry & Biochemistry have developed a new class of Histone Deacetylase (HDAC) inhibitors that can be tuned for isoform specificity and other properties.

New Molecular Tweezers Against Neurological Disorders And Viral Infections

UCLA researchers in the Department of Neurology with an international team of scientists have developed several new molecular tweezer derivatives with novel synthesis methods that significantly improved the therapeutic efficacy and pharmacokinetic characteristics of the drug candidates.

Antibody-Free Protocol For Generation Of Highly Expandable, Non-Fibroadipogenic Mesodermal Precursors from Human Pluripotent Stem Cells For Treatment

UCLA researchers in the Department of Orthopaedic Surgery have developed a novel method to generate mesodermal precursors from human pluripotent stem cells to treat chronic skeletal muscle atrophy and fibrosis.

Electrical Charge Balancing Scheme For Functional Stimulation Using Pulse Width Compensation

UCLA researchers in the Department of Bioengineering have developed a novel electrical charge cancellation scheme to effectively remove residual charge on an electrode, achieving greater precision for lesser hardware cost, while maintaining a surgically implantable small size without extra pulse insertion.

Protein Nanoparticles For Cancer Immunotherapy

Though new therapeutics for the treatment of cancer are constantly being developed, they often show low efficiency for long-term remission, adverse side effects, and low immune response. Scientists at UCI have found a way to combat these issues with a combination therapy delivered by nanoparticle of both a vaccine, to prime the immune system, and a checkpoint inhibitor to shut down anti-cancer immune responses. This has been shown to prolong survival and promote immune response and immunological memory related to long-term survival.

Transcription Factor Treatment for Schizophrenia and Bipolar Disorder

Current time to diagnosis for Schizophrenia and Bipolar Disorder is extremely lengthy (~6 months), and a delay in treatment greatly increases risk for suicidal thoughts. Once diagnosis has occurred, therapeutic options for both mental illnesses are greatly varied and have numerous side effects. To address both issues, UCI researchers have developed a novel way to diagnose and treat Schizophrenia and Bipolar Disorder by targeting specific transcription factors.

Viral Expression Vector Targeting HIV-1

UCLA researchers from the Department of Microbiology, Immunology, & Molecular Genetics have developed a viral expression vector that combines two reagents effective against HIV-1 infection.

Methods To Enhance Cancer Immunotherapy

Immune checkpoint inhibitors, such as antibodies that block negative regulators of T-cell activation (such as CTLA4 and PD-1/PD-L1), have transformed cancer treatment. However, even in metastatic melanoma and non-small cell lung cancer (NSCLC), malignancies that are highly responsive to immune checkpoint inhibitors (ICI), response rates rarely exceed 40%. Not only that, many common malignances, such as prostate cancer and pancreatic ductal adenocarcinoma, are ICI refractory but causes of treatment failure are largely unknown. It is known though that oxaliplatin triggers a form of cell death that is thought to be immunogenic, whereas the chemical analogue cisplatin does not trigger the same form of immunogenic cell death.   Recent clinical trials have shown that immune checkpoint inhibitors responsiveness is significantly augmented by combining PD-1 signaling inhibitors with platinoid chemotherapeutics. Such results have led to approval of immune checkpoint inhibitors + platinoid combination therapy in NSCLC but the basis for this synergism has not been determined.

Development of a New Biomarker for Diagnosis of Cardiovascular Disease: Monoclonal Antibody to Oxidized Cholesteryl Esters

Cardiovascular disease (CVD) is the leading cause of death and disability worldwide. The primary prevention of CVD is dependent upon the ability to identify high-risk individuals long before the development of overt events. This highlights the need for accurate risk stratification. An increasing number of novel biomarkers have been identified to predict cardiovascular events. Biomarkers play a critical role in the definition, prognostication, and decision-making regarding the management of cardiovascular events. There are several promising biomarkers that might provide diagnostic and prognostic information. The myocardial tissue-specific biomarker cardiac troponin, high-sensitivity assays for cardiac troponin, and heart-type fatty acid binding potential help diagnose myocardial infarction (MI) in the early hours following symptoms. Inflammatory markers such as growth differentiation factor-15, high-sensitivity C-reactive protein, fibrinogen, and uric acid predict MI and death and many others. However, there is a high unmet medical need for the more specific biomarkers that reflect different aspects of the development of atherosclerosis. 

Development of a Small Molecule that Blocks Alpha Synuclein Transmission in Neurodegenerative Disorders

There is a strong correlation with aging and the onset of developing a neurodegenerative disease such as Alzheimer’s or Parkinson’s disease, amyotrophic lateral sclerosis, frontotemporal dementia (FTD) and Creutzfeldt-Jacob disease, Dementia with Lewy Bodies (DLB), Multiple system atrophy (MSA) and others. A universal commonality among these diseases is the presence of misfolded aggregated proteins in the brain or with cells of the brain. Very strong evidence supports a role of spreading of misfolded proteins from cell to cell and across the brain in disease progression. Moreover, these aggregated proteins can take different forms and be used help diagnosis the specific neurodegenerative disease. Parkinson’s disease (PD) is characterized by loss of striatal dopaminergic signaling and the presence of alpha-synuclein-containing Lewy bodies and neurites. Research has shown the importance of alpha-synuclein (α-Syn) from examining people with PD at autopsy and the pathology associated with the disease which contains misfolded and aggregated α-Syn. Moreover, a mutation in the gene encoding α-Syn (SNCA) or simple overexpression of wild-type α-Syn will lead to PD. The misfolding and spread of α-Syn are central to disease initiation and progression. The presence of misfoided α-Syn is also seen in other synucleinopathy diseases including Alzheimer’s disease (AD) and Dementia with Lewy Bodies (DLB), the two most prevalent progressive dementia diseases and MSA. One of the most common forms of symptomatic treatment for early stages of PD is the use of monoamine oxidase B inhibitors and in later stages the use of dopamine receptor agonists (DRAs) and /or levodopa. The treatment must find a good balance between clinical benefits and risks. Ultimately, these treatments fail to show improvement over the course of 2-5+ years, therefore, new alternative treatments are needed especially those attacking the underlying course of the disease. Small molecule binding to native states of globular proteins has been successfully to block misfolding and aggregation most notably in the case of targeting transthyretin to treat systemic amyloidosis. By contrast, targeting of intrinsically disordered proteins such as native monomeric α-synuclein (α-Syn) with  small molecules has been challenging due to their inherent structural heterogeneity and the absence of persistent structural elements.

A High Potency CYP3A4 Inhibitor for Pharmacoenhancement of Drugs

      CYP3A4 is the most clinically relevant drug metabolizing enzyme in the body, as it is responsible for the oxidation and breakdown of ~60% of current drugs on the market.  Researchers at UCI have developed novel CYP3A4 inhibitors, that are highly potent and more specific, exhibit fewer side effects, and are both cheaper, and easier to-synthesize than current commercially available CYP3A4 inhibitors. 

Combination of a drug with low level light therapy (LLT) for treatment of wounds

This is a combination of a drug and light technology for the purpose of accelerating the healing of wounds on the skin, ulcers, and elsewhere in the body. Both methods have been shown to accelerate wound healing, and combining the two will potentially result in more rapid healing than either would alone.  

Immunotherapy For HIV/AIDS

Chronic HIV infection results in exhaustion and loss of the immune system, a phenomenon characterized by dysfunctional HIV-specific killer T cells. The exhausted T cells display inhibitory proteins on their surface, and scientists hope to be able to restore immune function by interfering with the negative signals transmitted by such proteins. PD-1, Lag-3 and Tim-3, some examples of T-cell exhaustion markers that are associated with immune activation. In fact, expression of multiple inhibitory receptors has been demonstrated to correlate positively with both plasma viral load and disease progression in HIV infected individuals. However, little is known about the development and maintenance, as well as heterogeneity of immune cell exhaustion. Another problem with chronic HIV infection is that a large number of people that are receiving antiviral therapy (ART) become resistant to treatment. 

Rational Design of Aminoglycoside-Based Antibiotics

UCLA researchers in the Department of Bioengineering have designed a novel class of antibiotics that are effective against resistant bacterial infections.

Trehalose Hydrogels For Stabilization And Delivery Of Proteins

UCLA researchers in the Department of Chemistry and Biochemistry have developed a novel trehalose hydrogel to help stabilize proteins for drug delivery.

Hydrogel For Engineered Immune Response

UCLA researchers in the Department of Chemical and Biomolecular Engineering have developed a novel biomaterial that can be used as a therapeutic for cancer, wound healing and other diseases.

Sustained Intracellular RNA Delivery and Expression

UCLA researchers in the Department of Chemistry and Biochemistry have developed a novel method for high protein expression levels, in situ, involving RNA-based therapeutics.

Systems And Methods For Therapeutic Agent Delivery

UCLA researchers at the Department of Physics have developed a system that is capable of delivering a therapeutic agent to a specifically targeted tissue using ultrasound.

Nanoparticles and Imaging Methods for MRI-Guided Stimuli-Responsive Theranostics

UCLA researchers from the Department of Medicine have developed novel nanoparticle and imaging methods for the MRI-guided targeted delivery of therapeutic agents.

Methods Comprising Immune System Modulation With Microporous Annealed Particle Gels

UCLA researchers have developed a novel microporous annealed particle (MAP) scaffold that acts as both a tissue growth scaffold and an immune modulatory system. The technology permits continuous, time-encoded, modulation of the immune system delivered injection/implantation of fabricated scaffold, comprised of the MAP gel technology.

Methods to Transport RNA into Mitochondria

Researchers at UCLA’s Jonsson Comprehensive Cancer Center, the Department of Chemistry and Biochemistry, and the Department of Pathology and Laboratory Medicine have uncovered a role for an essential cell protein, polynucleotide phosphorylase (PNPASE) in shuttling RNA into the mitochondria, the energy-producing “power plant” of the cell. This discovery sets the foundation for the development of long-term nucleic acid-based correction of mitochondrial-encoded defects through stem cells or other treatment vehicles.

Prospective Isolation Of Tumor-Reactive Cytotoxic CD4+ T Cells For Bladder Cancer Therapy

UCSF researchers have discovered a method for the isolation and expansion ex vivo of an endogenous population of bladder tumor-reactive cytotoxic CD4+ T cells that can be used to specifically and potently treat bladder cancer.

Methods for Inhibiting the Expression of MDM2 to Block Progression of Leukemia

RNA-editing proteins are an important class of proteins that regulate key steps in post-transcriptional RNA processing. One of the most common and best characterized is the Adenosine-to-Inosine editing (A-to-I editing) process. The cell translating machinery recognizes inosine as guanosine and A-to-I editing is accomplished by adenosine deaminase acting on RNA (ADAR) enzyme family that includes ADAR1, ADAR2, and ADAR3. While ADAR3 appears to inhibit ADAR2 editing within coding regions, ADAR1 edits primarily within double stranded RNA (dsRNA) loops formed by inverted primate-specific Alu repetitive elements. Atypical RNA editing can result in the alteration of non-coding RNAs such as miRNAs which can be present in different cancers and play a role in their development.  

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