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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.

Scientists at UCSF and the Parker Institute of Cancer Immunotherapy have developed methods for characterizing dendritic cells as well as methods for identifying a dendritic cell as either an inflammatory or a tolerogenic dendritic cell. Their results provide important insights into previously obscured metabolic heterogeneity impacting immune profiles of immunogenic and tolerogenic dendritic cells (DC).

Researchers at UCSF have developed methods of treating Rheumatoid arthritis and for predicting the response of patients to methotrexate. 

Researchers at UCSF and the Chan Zuckerberg Biohub have identified multiple common autoantibody targets in APS1 patients through proteome-wide programmable phage-display.

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Researchers at UCSF have developed a method to selectively clear senescent cells by stimulating an immune response. Accumulation of senescent cells underlies a number of disease conditions and age-related pathologies. Current approaches to clear this cell type use senolytics, these are small-molecules that induce cell death of the senescent cells. Unfortunately, these compounds are not truly specific and affect other non-pathogenic cells. UCSF researchers eliminate these off-target effects by utilizing the body’s immune system to selectively target senescent cells for clearance. They do this by activation and expansion of certain immune cells. Stimulating the immune system to clear these cells is unprecedented in the field and offers a new therapeutic modality to treat senescence associated conditions. The technology has been fully validated in a laboratory setting.

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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.  

Small molecule pendrin inhibitors for treating inflammatory lung diseases.

A novel method to measure airway mucus plugging using CT images from patients with asthma or chronic obstructive pulmonary disease (COPD) patients.

UCSF researchers have developed a novel gut microbiome-based diagnostic test and targeted treatment for early-life identification of atopy or asthma risk in children.

A novel approach for the treatment of lung inflammatory and fibrotic diseases by increased or repaired chitinase function in lung tissues

This invention comprises compositions and methods for treatment of neuromyelitis optica (NMO) spectrum disorders using anti-aquaporin-4 (AQP4) antibody lacking effector function.

This invention proposes the first pharmacological treatment to prevent the onset of Type I diabetes by preventing beta cell destruction. 

UCSF researchers have invented a novel method to generate covalent macromolecular inhibitors. This strategy allows a peptide inhibitor to bind to its target protein specifically and irreversibly through proximity-enabled bioreactivity.

This technology contains a method for modulating the activity of the nuclear receptor LRH-1 with identified small molecule compounds that may be developed to treat inflammatory bowel disease, Type II diabetes, triple negative breast cancer and pancreatic cancer.

The Human Immunodeficiency Virus (HIV) has evolved a number of mechanisms of evading the human immune system.  One way is through a high level of mutation, which makes it difficult to develop a vaccine that stimulates protective immunity against all of the different HIV variants.  Therefore, scientists are searching for a general surrogate maker that could be used to target any HIV-infected cell regardless of its mutational status. In this regard, scientists have recently focused their attention on so-called cryptic peptides of HIV.  Cryptic peptides are non-functional HIV proteins that are produced due to translational errors that occur in HIV-infected cells.  Because these cryptic peptides are commonly produced and then presented on the surface of the HIV-infected cells, it is thought they may be good surrogate markers and targets for any HIV-infected cell.

Human Immunodeficiency Virus (HIV) has evolved a number of mechanisms of evading the human immune system.  One way is through a high level of mutation, which makes it difficult to develop a vaccine that stimulates protective immunity against all of the different HIV variants.  Therefore, scientists are searching for a general surrogate marker that could be used to target any HIV-infected cell regardless of its mutational status. In this regard, scientists have recently turned their attention to the APOBEC machinery in HIV cells.  APOBEC proteins are human proteins that modify genetic material of viruses so that they are unable to produce proteins essential for viral survival.  Remarkably, HIV evades the APOBEC defense by making a protein called Vif that re-routes APOBEC proteins to proteosomes for destruction thereby reducing APOBEC's protective functions.  However, this activity also increases the presentation of APOBEC antigens or peptides on the cell's surface. APOBEC peptides may be good candidates for surrogate HIV markers simply because they are present on the surface of all HIV-infected cells.  In addition, in order for HIV-infected cells to stop displaying APOBEC peptides on their surface, the virus would need to evolve mutations in the region coding for the Vif protein that re-routes the APOBEC proteins.  This would make the virus vulnerable to the defenses mediated by the functional APOBEC proteins.  This phenomenon should result in dual pressure on the virus that should slow or prevent the evolution of viral resistance to these T-cell responses.

Interstitial lung disease (ILD) is a common manifestation of systemic autoimmune diseases such as rheumatoid arthritis (RA), lupus and scleroderma, which can lead to inflammation and scarring of the lung and, consequently, to hypoxemia, pulmonary hypertension and death.  It is estimated that ILD occurs in approximately 15 percent of patients with RA.  Very little is known about how ILD disorders arise and what role loss of immune tolerance plays in ILD development.  Presently, there are no validated lung-specific autoantigens for diagnosis of autoimmune-mediated lung disease.  Current options for ILD treatment are limited to powerful immunosuppressive medications with significant side effects.  Identification of novel pulmonary biomarkers is sorely needed to develop better diagnostic methods and therapies for ILD.

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