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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.

A Novel Method For The Treatment Of Social Dysfunction Associated With Autism Spectrum Disorder (ASD) By Activating Dopamine Receptor 3 (Drd3) Signal

Children exposed to early life stress (ELS) such as physical abuse and emotional neglect during a critical period in their development are more likely to display social dysfunction later in life. Symptoms of disrupted social behaviors include decreased social motivation and a lack of interest in attending to social stimuli or seeking and enjoying reciprocal social interactions. Given that high levels of early adversity are also associated with the asocial behaviors of psychiatric patients, identifying the neural mechanisms underlying ELS-induced social dysfunction is essential for the development of treatment strategies for mental illnesses associated with social impairments, such as autism spectrum disorder (ASD), schizophrenia, and major depressive disorder (MDD).

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.

Integrin Stimulating Materials For The Normalization Of Diseased Vasculature

UCLA researchers in the Department of Chemical and Biomolecular Engineering have developed a novel a novel α3/α5β1 integrin binding site integrated endothelial growth factor (VEGF) delivery hydrogel that directs therapeutic vessel regeneration and reduces VEGF induced vascular permeability.

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.  

A Novel Method to use tRNA Manipulation to Treat Tumors and Viral Infections

DNA damaging agents (DDA) have been successfully used therapeutically to treat an extensive range of solid tumors and blood cancers including lymphomas and leukemias. Despite its broad use, many tumors become resistant to DDA therapies over the course of the treatment. One strategy to overcome this shortcoming is the ability to reverse the resistance to DDAs. The Schlafen (SLFN) gene family members encode a diverse group of proteins, which play important roles in regulating biological functions including cellular proliferation, immune responses and suppression of viral replication. Some SLFN family members have been reported to inhibit growth in cancer cells and promote cancer cell sensitivity to chemotherapeutics. Human SLFN11 (Schlafen 11) is one such member whose expression deficiency has been observed to cause chemotherapeutics resistance in a vast collection of tumor cells. SLFN11 sensitizes tumor cells to DNA damaging agents (DDA) by preferentially inhibiting protein syntheses of multiple components of the DNA damage response and repair pathways (e.g. ATR and ATM). Alternatively, tumor cells might not only acquire drug resistance from loss of Slfn11 during treatment, but might already be drug-resistant and Slfn11-deficient a priori. Unlike those highly expressed housekeeping genes, the proteins syntheses of most genes involved in DNA damage response and repair heavily rely on tRNA-Leu-TAA (for leucine), which is cleaved by SLFN11 upon DNA damages. The inventors also have shown previously that SLFN11 inhibits HIV replication by inhibiting syntheses of viral proteins through a similar mechanism. As such, the destruction of tRNA-Leu-TAA (e.g. by antisense oligonucleotides Gapmer) or their inactivation (e.g. via leucine tRNA synthase inhibition) inhibits DNA damage repair and restores the sensitivity of DDA-resistant tumor cells to these chemotherapeutic agents, and can also be used as a novel approach to target viruses that rely on tRNA-Leu-TAA for their replication.

PAK4 in Cancer Immune Exclusion

Researchers at the UCLA David Geffen School of Medicine have proposed that T cell tumor infiltration and the anti-tumor activity of anti-PD-1/PD-L1-based immunotherapy to be modulated through kinase PAK4 activity.   

Combination Therapy Approach Using Novel Biguanides For Cancer Treatment

Researchers in the UCLA Departments of Molecular and Medical Pharmacology, Chemistry and Biochemistry, Surgery, and Medicine have developed novel metformin analogues which, when combined with immune checkpoint inhibitors, enhance the therapeutic benefit of these inhibitors in treating triple-negative breast cancer and other malignancies.

Method To Determine Personalized Transcranial Magnetic Stimulation (Tms) Parameters To Enhance Clinical Treatment Outcomes In Major Depression And Neurological Disorders

Researchers led by Aimee Hunter from the Department of Psychaitry at UCLA have developed a methodology to determine parameters for personalized transcranial magnetic stimulation to treat depression.

Chemoselective Side-Chain Modifications Of Methionine-Containing Elastin-Like Polypeptides

UCLA researchers in the Department of Bioengineering and Department of Chemistry & Biochemistry have developed a novel method for the introduction of various functional groups onto recombinant elastin-like polypeptides (ELPs), creating new compositions of ELPs that may be used for medical therapeutic or diagnostic applications.

Method And Kits For Identifying Treatment Targets Of Cancer

Tumorigenesis is a multistep process involving genetic alteration and gene expression deregulation in cells. Over the past few decades, targeted therapies hold hope for the treatment of many types of cancer. A common complication is that cancer drugs eventually stop working owing to the tumor heterogeneity and the genetic complexity of the tumor. Previous studies using pharmacological, RNA interference or CRISPR-mediated screens have enabled target identification, however, many targets genes cannot be further validated in vivo due to the lack of understanding of their corresponding signaling and gene network or there is biased selection due to over emphasis on particular phenotypes such as growth or depletion of cancer cells.    UC Berkeley researchers have developed a platform using molecular feature recognition and CRISPR-based target interrogation, in order to explore gene regulatory networks for new drug target identification and validation.  One aspect of the technology relates to a method for identifying treatment targets relating to tumors. 

Composition of NY-ESO-1-Specific T Cell Receptors Restricted on Multiple Major Histocompatibility Complex Molecules

Researchers at the UCLA Department of Microbiology, Immunology, and Molecular Genetics have isolated T cell receptors (TCRs) specific for NY-ESO-1 epitopes presented by four MHC molecules, and developed novel approaches to extend TCR gene therapies targeting NY-ESO-1 using these TCRs.

Modulating IRE1a/ß Kinase for Treatment of Unfolded Protein Response (UPR)-related Diseases

This invention identifies a series of compounds which can selectively regulate the kinase activity of IRE1α and IRE1β, which are paralogous enzymes critical for the activation of the unfolded protein response (UPR) and that may have implications in cell-degenerative diseases such as diabetes, cancer, fibrosis, asthma, and retinitis pigmentosa.  

Novel Cyclic Dinucleotide Analogues as STING Agonists

Stimulator of interferon genes (STING) is known to be a central mediator of innate immunity. It is a 379 amino acid protein expressed in various endothelial and epithelial cell types as well as in hematopoietic cells such as T cells, macrophages and dendritic cells. STING is naturally activated by aberrant DNA species via formation of native cyclic dinulcleotides (CDNs) in cytosol of the cell. When stimulated STING induces the expression of type I interferon (IFN), cytokines and T cell recruitment factors that result in the activation of macrophages and dendritic cells, innate effector cells such as natural killer (NK) cells and priming of tumor specific T cells. Recent studies have shown that the STING pathway is essential for radiation induced and spontaneous natural antitumor T cell responses. Tumor cells often induce an immunosuppressive microenvironment favoring cancer development. Targeting STING pathway by using TING agonists to produce IFNs for enhancing antitumor immune response may provide an alternative strategy for the improvement of cancer immunotherapy.

Method And Substance For The Treatment Of Cerebral Amyloid Angiopathy

UCLA researchers in the Department of Pathology have identified a novel pathway that causes β-amyloid-associated smooth muscle cell loss in Cerebral Amyloid Angiopathy (CAA) cases.

Targeting Unique Copy Number Vulnerabilities In Tumor Cells

In the last ten years much progress has been achieved on the genome analysis of cancer causing genes, mainly driven by the advances of whole genome sequencing and the identification of driver mutations. The detection of thousands of mutations in single samples as well as in large cohorts led by The Cancer Genome Atlas (TCGA), the Cancer Genome Project (CGP), and the International Cancer Genome Consortium have driven these advances. As of 2018, data from these projects and current literature has generated a list of 522 cancer genes in the Catalogue of Somatic Mutations in Cancer (COSMIC). The increasing application of precision medicine for individual cancers has increased the focus of identifying additional and rare drivers of mutations. To that end, some focus has been on the identification of oncogene amplification on extrachromosomal DNA.

Combination Immunotherapies for Treatment of Liver Cancer

Primary liver cancer, with the majority being hepatocellular carcinoma (HCC), is now the second leading cause of cancer mortality and the fifth most common cancer worldwide, claiming approximately 800,000 life every year. HCC is a chemotherapy resistant tumor with limited treatment options including surgical resection, liver transplantation and local ablation at the early stages. Sorafenib, a multi-kinase inhibitor, remains a first-line systemic drug for advanced HCC even with poor outcomes, and similar low therapeutic benefits were reported for regorafenib, lenvatinib, and cabozantinib. Over 100 clinical trials that tested other compounds or approaches have failed to show therapeutic benefit to HCC patients. Immunotherapy by blocking inhibitory pathways in T lymphocytes, such as the PD-L1/PD-1 axis, is being widely tested in various solid tumors. Notably, this emerging therapeutic approach is already in clinical trials for advanced HCC in multi-centers around the globe. Two latest reports on open-label, non-randomized, phase 1/2 trials with pembrolizumab or nivolumab indicated manageable safety in advanced HCC patients with or without prior sorafenib treatment, albeit with very limited therapeutic benefits observed so far. The outcome of immunotherapy for liver cancer can be compounded by the unique immunotolerant microenvironment in the liver. A variety of clinical trials are ongoing to evaluate combination of immune checkpoint inhibitors or with other drugs, without clear justification or support by preclinical data.

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