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Lysosomal Protein Targeting Sequence and Therapeutic Applications
Researchers have identified a GCase protein with a specific mutation that increases the protein’s binding in the lysosome. Inside the lysosome there is a receptor called “Lysosomal Integral Membrane Protein 2” (LIMP-2). This receptor is the “docking station” for the GCase protein. Lysosomal storage disorders and neurological...
Published: 3/15/2024
Category(s): Therapeutics
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Compounds for Therapeutic Intervention of Neurofibroma type 1
Compounds for Therapeutic Intervention of NF Type 1(CHMC Ref. Id: 2007-0702)Overview: Loss of function mutations in the Neurofibromatosis type 1 (NF1) gene result in an autosomal dominant disease that affects approximately 1 of every 3500 live births. Ninety-five percent of patients develop neurofibromas, benign Schwann cell tumors that can associate...
Published: 8/20/2018
Category(s): Therapeutics
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Therapeutic Prevention of Obesity in At-Risk Populations
Identifying safe/effective methods of regulating obesity associated weight gain has been problematic. Our technology represents methods/compositions for weight regulation and energy metabolism. We identified ligands, B-cell activating factor (BAFF), a proliferation-inducing ligand (APRIL) or their modulators, that prevent obesity. Animals expressing...
Published: 3/13/2026
Category(s): Therapeutics
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MAPK Inhibition for Pulmonary Fibrosis
Published: 8/21/2018
Category(s): Therapeutics
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Method for Treating Acute Kidney Injury
This invention is based, at least in part, on the discovery that kidney injury results in an up-regulation of Wnt ligands in macrophages and the canonical Wnt response in epithelial cells. While macrophages from the injured kidney are a source of increased Wnt activity, compromise of Wnt receptors or conditional deletion of Wnt7b in the macrophage lineage...
Published: 6/16/2025
Category(s): Therapeutics
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Application for MR-Guided Focused Ultrasound
Application for MR-Guided
Focused Ultrasound
(CHMC Ref. Id: 2009-0405)
Overview:
This technology provides systems and methods for using Magnetic
Resonance (MR) image-guided delivery of focused ultrasound energy to reduce
the amount of visceral fat in a patient as a therapeutic for the treatment of
multiple disease conditions linked to the accumulation...
Published: 8/20/2018
Category(s): Therapeutics
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Therapeutic Prevention of Obesity
Identifying safe and effective methods of regulating obesity associated weight gain is problematic. This technology represents a novel method for treating or preventing diet-induced obesity and one or more of its sequelae by inhibiting RP105 (radioprotective protein 105kDa) expression and/or MD-1 expression. Researchers demonstrated that RP105 deficient...
Published: 12/11/2025
Category(s): Therapeutics
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Critical Role for Eotaxin-3 in Eosinophilic Esophagitis
Critical Role for Eotaxin-3
in Eosinophilic Esophagitis
(CHMC Ref. Id: 2004-1003)
Overview:
Eosinophilic esophagitis (EE) is often confused with gastroesophageal reflux
disease (GERD), but it does not typically respond to anti-GERD therapy.
Patients with EE typically have elevated levels of eosinophilis in their
esophageal tissue and diagnosis...
Published: 8/28/2025
Category(s): Therapeutics
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C5a Receptor Antagonist for Treatment of Inflammatory Diseases
The anaphylatoxin C5a, part of the complement system, is involved in the pathogenesis of systemic inflammatory diseases such as sepsis, allergic asthma, adult respiratory distress syndrome and rheumatoid arthritis. C5a is an excellent therapeutic target in inflammatory diseases and blocking the C5a receptor with a C5a receptor antagonist is an ideal...
Published: 6/20/2023
Category(s): Therapeutics
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Pharmaceutical Targeting of Eosinophils by CCR-3 Mediated Internalization
Pharmaceutical Targeting of
Eosinophils by CCR-3 Medicated Internalization
(CHMC Ref. Id: 1999-0210)
Overview:
Eosinophils normally appear in peripheral blood at a concentration of ~1-3%
of total leukocytes and their presence in tissue is primarily restricted to the
mucosa. However, in various disease states, such as eosinophilic esophagitis
(EoE),...
Published: 8/23/2021
Category(s): Therapeutics
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