A high-throughput platform for the non-invasive intracellular delivery of molecules for therapeutic applications

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    Isolating live cells after high-throughput, long-term, time-lapse microscopy
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A natural beta interferon-inducing therapeutic glycolipid from the microbiome that prevents and treats severe viral infection

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    Methods for modulating TLR4 signaling
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A novel biologic and therapeutic target for the treatment of diabetes

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    Secreted protein that affects blood glucose levels
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Application of the SPLANDID antigen presentation technology to HIV-1 vaccine development

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    Membrane protein nanoparticles for antigen presentation
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Assessment of targeted protein degradation as a strategy for potent antivirals with high barriers to resistance

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    Degraders of Hepatitus C Virus NS3/4A Protein
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Building better screens and identifying ligands for human MS4A proteins

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    Identification of the MS4A family as ligand-gated ion channels
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Computationally designed therapy targeting lysosomal function and inflammation in neurodegenerative diseases

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    Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
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    Antisense oligonnucleotide drug targets
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Developing novel therapeutic approaches for CNS drug delivery by targeting a newly identified key regulator of the blood brain barrier

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    Methods and compositions relating to modulation of the permeability of the blood brain barrier
    (Abandoned)
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    Structure and mechanisms of the blood-brain barrier DHA transporter Mfsd2a
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    Methods and compositions relating to treatment of CNS diseases
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Development of AAV-mediated mini-gene therapy for Usher syndrome type 1F, a combined deafness and blindness

  • WO2020219990
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    Mini-PCDH15 for treatment of deafness and blindness
    (Patent application)

Development of an inexpensive at-home influenza kit and detection technology

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    :
    Enhancement of RT-RPA reaction by the addition of RNaseH
    (Abandoned)
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    :
    Development of an inexpensive at-home influenza
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Development of small molecule modulators of autophagy and ER stress as novel therapeutics for AD

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    Compounds and methods for blocking apoptosis and inducing autophagy
    (Patent application)

Development of synthetic analogs of a sleeve gastrectomy-induced metabolite as a novel therapy for type 2 diabetes and obesity

  • WO2020041673
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    Compositions and methods related to cholic acid 7-sulfate as a treatment for diabetes
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    Methods for inducing bile acid sulfotransferase SULT2A for treating metabolic disorders
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    Synthetic derivatives of cholic acid 7-sulfate and uses thereof
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Engineered proteins for treatment of pancreatitis

Evaluating splicing modulators for therapeutic impact in cohesin-mutant AML and MDS cells

  • WO2023147400
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    Compositions and Methods for Inhibiting STAG1 Expression and Uses Thereof
    (Patent application)

Evaluating splicing modulators for therapeutic impact in cohesin-mutant AML and MDS cells

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    :
    Compositions and methods for inhibiting STAG1 expression and uses thereof
    (Patent application)

Gene therapy for the treatment of Wolfram Syndrome II

  • WO2021222476
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    DAEUS: a high effciency gene delivery system for overexpression of geroprotective genes
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    High efficiency purification of divergent AAV serotypes using AAVX affinity chromatography
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Identification of small molecule binders of the novel cancer immunotherapy target Bird33

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    Methods and compositions for treating cancers by modulating the expression and/or activity of STUB1
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Identifying novel therapeutic targets for treating pain and chronic itch

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    G-protein-coupled receptors (GPCRs) regulating; somatosensation from single-cell transcriptome atlas
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Isoguvacine and benzodiazepine derivatives for the treatment of tactile hypersensitivity and anxiety in autism spectrum disorders

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    Methods and compositions for reducing tactile dysfunction and anxiety associated with autism spectrum disorder, Rett syndrome, and Fragile X syndrome
    (Patent application)
  • WO2019232046
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    Compositions and methods for reducing tactile dysfunction, anxiety, and social impairment
    (Patent application)
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    Compositions and methods for reducing tactile dysfunction, anxiety, and social impairment
    (Patent application)
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    Compositions and methods for reducing tactile dysfunction, anxiety, and social impairment
    (Patent application)
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    Human tactile prepulse inhibition assay
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Manipulating mitobiogenesis by targeting ME2

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    Compositions and Methods for Modulating Mitochondrial Function and Biogenesis
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Methods and applications of semi-permeable capsules with current application to high-throughput single cell multi-omics

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    Core-shell capsules and uses thereof
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Multiplex editing the human genome

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    Highly multipexed genome editing
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Next-generation AAV therapies for management of intractable chronic pain

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    A scalable platform for the development of cell-type-specific viruses
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    Nociceptor-specific gene regulatory elements for the treatment of pain
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Small molecule modulators of gut bacterial bile acid metabolism to treat metabolic syndrome and associated non-alcoholic fatty liver disease (NAFLD)

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    Small molecule modulators of gut bacterial bile acid metabolism
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Studies on immunogenic lipids from the gut microbiome

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    Immunomodulatory lipids and uses thereof
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Targeting apoptotic pathways for ALS, Alzheimer’s Disease and Parkinson’s Disease

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    Ion channel binders and uses thereof
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Targeting neuro-immune signaling to treat bacterial infections

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    Methods and compositions for treating a microbial infection
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