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Cas12a System For Combinatorial Transcriptional Repression In Eukaryotic Cells
The invention is a novel system for delivering combinatorial transcriptional silencing. Conventional approaches using Cas12a for … moreThe invention is a novel system for delivering combinatorial transcriptional silencing. Conventional approaches using Cas12a for transcriptional silencing rely on transient transfection experiments where both protein and guide RNA (gRNA) components are highly overexpressed. This system can function under low ribonucleoprotein expression to enable combinatorial targeting of multiple genomic sites per cell in high-throughput sequencing based pooled screens and therapeutic delivery in vivo. less
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Feb 14, 2024
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Novel therapy for inflammatory disease using fatty acid-bound alpha fetoprotein
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Sep 28, 2023
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Novel compositions and methods for targeted replacement of endogenous T-cell receptor with a chimeric antigen receptor
This invention is a method for targeted and high-efficiency replacement of the endogenous T‐cell receptor (TCR… moreThis invention is a method for targeted and high-efficiency replacement of the endogenous T‐cell receptor (TCR) with a chimeric antigen receptor (CAR) by introducing a Cas9 and guide RNA (gRNA) ribonucleoprotein (RNP) that targets a genomic break to the endogenous T‐cell receptor alpha constant chain (TRAC) locus. The depletion of TCR‐positive cells by magnetic beads enriches the cells which have both the CAR knockin and TCR knockout. Also cells, which are modified successfully by homology‐directed‐repair (HDR) through minimizing non‐homologous‐end‐joining (NHEJ)‐mediated TCR knockout by the selection of gRNA targets that do not disrupt protein‐coding regions, are enriched. A set of HDR templates, which use recombinant adeno‐associated virus (rAAV) or ssDNA/dsDNA Cas9 “shuttle” hybrid templates, is adapted along with selected gRNA targets that stimulate high-efficiency HDR with reduced disruption of endogenous TCR expression in the absence of HDR. less
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Jul 19, 2023
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Gene Targets For Gamma-Delta T Cell Cytotoxicity Against Tumor Cells
Using a genome-wide knockout screen in target tumor cells, UCSF Investigators have identified cellular factors that… moreUsing a genome-wide knockout screen in target tumor cells, UCSF Investigators have identified cellular factors that influence gamma-delta T cell cytotoxicity against target cells, and developed methods for modulating expression of these factors. One of the top genetic hits is of particular importance since it is a cell surface protein that has not been previously implicated in this interaction. These methods have great therapeutic potential and may lead to treatments for a variety of diseases, including cancer, autoimmune diseases, bone disorders, metabolic disorders, and infectious diseases. less
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Jul 19, 2023
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Gene Targets For Manipulating T Cell Behavior
By performing non-viral pooled knock-in screens, UCSF investigators have discovered novel genes that improve T cell … moreBy performing non-viral pooled knock-in screens, UCSF investigators have discovered novel genes that improve T cell functionality across a variety of in vitro assays. This invention includes novel compositions and methods for modifying the genome of a T cell to alter its specific and functionality, while limiting the side effects associated with T cell therapies. Technology Advantages: - Identifies novel gene targets
- Potential to enhance therapeutic potency of T cells for cancer and autoimmune applications
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Jul 19, 2023
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METHOD FOR MANUFACTURING THERAPEUTIC IMMUNE CELLS
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Jul 15, 2023
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INTEGRATED OPTICAL PEEL AWAY PLEUROSCOPIC TROCAR FOR USE IN PLEUROSCOPIC AND THORACIC PROCEDURES
The invention is a novel trocar device for pleuroscopic and thoracic procedures which simplifies trocar placement … moreThe invention is a novel trocar device for pleuroscopic and thoracic procedures which simplifies trocar placement. This trocar will also serve as the pleuroscope itself. Trocars are required for any form of video assisted thoracic surgery, including medical thoracoscopy. Pleuroscopic procedures such as these are performed when patients develop gas or liquid in the pleura and allow full chest wall examination, pleural biopsies, washouts in cases of infection or bleeding, lysis of adhesions or chest tube placement. Conventional approaches require blunt dissection of the chest wall, which is invasive and painful for the patient, and requires a higher level of standardized training to perform the procedure which limits the utilization of this procedure. The novel trocar has the potential to replace pleuroscopes in wide variety of indications while extending its use to a wider physician pool. The device will allow easier access to pleuroscopy equipment, reduced overhead with regards to gear purchasing and maintenance, facilitate biopsies, impact pleural infection management and improve directed chest tube placement. To address these current challenges, a disposable plastic trocar that enables minimally invasive single incision procedures for use in pleuroscopic and thoracic surgeries has been developed. less
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Jun 22, 2023
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New Generation Bitopic Bcr-Abl Inhibitors
UCSF researchers have synthesized two classes bitopic inhibitors of BCR-ABL that have been verified for … moreUCSF researchers have synthesized two classes bitopic inhibitors of BCR-ABL that have been verified for their biochemical activity against ABL1 kinase domain and validated for their ability to specifically inhibit BCR-ABL signaling in cells. Further optimization can be done in both the ligand and linker components and additional biological validation will be needed prior to clinical trial. less
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Jun 14, 2023
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High-Throughput Discovery Of Bipartite Or Tripartite Crispr-Based Epigenetic Editors
The invention is a novel platform for identifying and characterizing programmable gene modulators (PGM). The epigenome … moreThe invention is a novel platform for identifying and characterizing programmable gene modulators (PGM). The epigenome editor discovery platform has potential utility in both therapeutic development/discovery and as a research tool.
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Primary:
University of California, San Francisco (UCSF)
Date posted:
May 13, 2023
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NOVEL OPIOID RECEPTOR AGONIST FOR ANALGESIA WITH REDUCED SIDE EFFECTS
Researchers at the University of California San Francisco, in collaboration with Stanford University, the University … moreResearchers at the University of California San Francisco, in collaboration with Stanford University, the University of North Carolina at Chapel Hill and Friedrich Alexander University in Erlangen, Germany, have identified a novel molecule that acts as an agonist for the µ opioid receptor (MOR) with Ki of 3.8 nM. Importantly, this molecule efficiently activates the G-protein pathway, which mediates the analgesic effects, with only modest activation of the arrestin pathway, which is responsible for many of the side effects attributed to opioids. Increased pain tolerance in vivo has been confirmed in multiple mouse studies by two different laboratories. The molecule does not confer respiratory depression compared to vehicle and substantially reduces the level of constipation experienced with equipotent doses of morphine. Early mouse studies suggest less predilection towards addiction. The molecule does is 1000-fold specific as an agonist for the MOR vs other opioid subtypes and against the hERG ion channel, and 10,000-fold specific against the transporters NET, SERT, and DAT. less
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Primary:
University of California, San Francisco (UCSF)
Date posted:
Apr 19, 2023
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