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Cas12a System For Combinatorial Transcriptional Repression In Eukaryotic Cells
Brief description not available
Novel therapy for inflammatory disease using fatty acid-bound alpha fetoprotein
α-Fetoprotein (AFP) is a fetal glycoprotein produced by the majority of human hepatocellular carcinoma tumors and other tumor types. Delineating differences between fetal 'normal' AFP (nAFP) and tumor-derived AFP (tAFP), investigators at UCSF and the Parker Institute for Cancer Immunotherapy have uncovered a novel role for tAFP in altering metabolism via lipid-binding partners. They have developed a pharmaceutical composition comprising AFP bound by a fatty acid which, depending on the fatty acid used, can have an immunosuppressive effect allowing for the treatment of inflammatory diseases. AFP bound to other fatty acids can eliminate the immune suppressive impact and have a neutral effect which allows for the development of dendritic cell (DC) vaccines presenting AFP epitopes which could be used to treat and prevent tumor AFP-expressing cancers.
Novel compositions and methods for targeted replacement of endogenous T-cell receptor with a chimeric antigen receptor
Gene Targets For Gamma-Delta T Cell Cytotoxicity Against Tumor Cells
Gene Targets For Manipulating T Cell Behavior
METHOD FOR MANUFACTURING THERAPEUTIC IMMUNE CELLS
Chimeric antigen receptor (CAR) T cells have so far shown limited efficacy on brain and solid tumors. UCSF investigators have developed a method of manufacturing recombinant immune cells by pre-treating them with a combination of small molecules to increase the number of CAR T cells in the tumor microenvironment and improve the survival of animal models bearing glioma in the brain relative to CAR T cells that have not received the pre-treatment. These results may be applicable to other solid tumors.
High-Throughput Discovery Of Bipartite Or Tripartite Crispr-Based Epigenetic Editors
Currently, conventional methods of epigenome editor discovery require time and labor-intensive construct development, which is typically performed in low-throughput arrayed formats. The platform bypasses current time/labor constraints (and without reliance on construct barcodes) to facilitate the identification of an optimal gene modulator in a single experiment.
LONG NON-CODING RNAS (LNCRNAS) AS THERAPEUTIC TARGETS IN GLIOMA
Novel Cell Therapy for CTLA4 Haploinsufficiency
Scientists have developed a CRISPR-Cas9 based genome editing method for universal correction of disease-causing mutations in the CTLA4 gene, which most commonly manifest as a Primary Immunodeficiency. Current treatment involves monthly IV injections or weekly subcutaneous injections of a recombinant CTLA4-Ig fusion protein abatacept. This invention includes one-time infusion of a CTLA4-corrected autologous T cell therapy. The corrected patient cells are generated by ex vivo electroporation of a specific gRNA:Cas9 ribonucleoprotien (RNP) complex and cognate homology-directed-repair template (HDRT) targeting a functional copy of the CTLA4 gene within an intronic region of the endogenous CTLA4 gene. This combination allows for (1) highly efficient knockin (up to 70% in patient cells), (2) cell-type and context specific regulation of CTLA4 expression under natural promoter and regulatory elements, and (3) preservation of endogenous CTLA4 expression in uncorrected cells.
Therapeutic For Dry Age-Related Macular Degeneration and Stargardt Disease
Targeting Piezo1 To Treat Inherited And Age-Related Macular Degenerations
Humanized Anti-Integrin aVß8 mAb to Inhibit TGFß Activity and Enhance Anti-Tumor Immunity for Immunotherapy
CRISPR-Cas Inhibiting Polypeptides
PHAGE-MEDIATED DELIVERY OF GENES TO GUT MICROBIOME
Researchers at UCSF have developed methods to engineer bacteriophage for gene delivery to gut microbiome.
ENGINEERED ACE2 RECEPTOR TRAPS TO BLOCK SARS-COV-2 INFECTION
Researchers at UCSF and the Chan Zuckerberg Biohub have developed a set of ACE2 variants which potently block SAR-CoV-2 infection in cells.
Improving primary human NK cell expansion with a chimeric cytokine receptor
Natural Killer (NK) cells are innate lymphocytes with the ability to lyse tumor cells. One limitation of NK cells when encountering tumor cells is that they can’t control their own proliferation and expansion to increase their numbers at the tumor site. Current approaches to increase NK cell numbers and stimulate NK-cell anti-cancer functions include systemic administration of recombinant cytokines (IL-15, IL-2, or IL-12) that exhibit systemic or local toxicity or constitutive expression of IL-15 in transduced NK cells. Researchers at UCSF have engineered NK cells with a chimeric cytokine receptor (CCR) that provides autocrine signaling through the secretion of IFNγ, which subsequently enhances NK cell proliferation and function to support NK cell anti-cancer immune response specifically at the tumor site while avoiding recombinant cytokine- related toxicity.
Cyclic Peptide Ligands of the Oncoprotein KRAS (G12D)
HTR1F Antagonists for Treatment of Diabetes and Improvement of Peri-Transplant Beta Cell Survival and Function for Type 1 Diabetes
Methods for Culturing Glioblastoma Cancer Cells and for Inhibiting Invasion of Cancer
Low-Intensity Pulsed Ultrasound (LIPUS) for Enhanced Testosterone Production and Treatment of Low Testosterone
An Extracellular Vesicle Based Liquid Biopsy for Treatment Monitoring and Early Detection of Tumor Recurrence in Glioblastoma Patients
Improved Small Molecule Activators Of K2p Potassium Channels
Integrin Activation to Bypass the CD47 Macrophage Checkpoint and Enable Phagocytosis of Cancer Cells
ELONGATION FACTOR 1-ALPHA INHIBITORS FOR THE TREATMENT OF MULTIPLE MYELOMA / MYC-DRIVEN CANCERS
Coordinately-Regulated Retroviral Gene Delivery System