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Novel Endoglycosidase Derivatives Treating Diseases of the Adaptive Immune System
Application
Protein-based therapeutics for the treatment of IgG-mediated diseases.
Key Benefits
A new class of endoglycosidases for the treatment of IgG-mediated diseases.
Four novel compounds have been developed that exhibited robust in vitro activity.
Potential to become first-in-class medications for dysregulation of the adaptive immune system,...
Published: 11/20/2024
Contributor(s): Eric Sundberg, Diego Sastre, Jonathan Du, Jeffrey Ravetch, Stylianos Bournazos
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Whole-Animal Genetic Model to Mimic Aspects of the Patient Phenotype of Classic Galactosemia
Application
An animal model for studying the underlying causes of galactosemia and screening for novel drugs to treat the condition.
Key Benefits
The first animal model for elucidating the underlying causes of galactosemia in infants.
Animals with galactosemia causing genetic knockout exhibit similar disease phenotypes and complications...
Published: 7/30/2024
Contributor(s): Judith Fridovich-Keil
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HDAC Inhibitors for Managing Vascular Conditions and Diabetic Peripheral Neuropathies
Application
Treatment of diabetic neuropathy using epigenetic modulators, histone deacetylase inhibitors (HDACis), to ameliorate or prevent vascular conditions and neuropathic pains by nerve damages.
Key Benefits
Reverses or protects against diabetes-induced nerve damages by direct injection of HDACi.
Relatively low regulatory risk because many...
Published: 11/20/2024
Contributor(s): Young-Sup Yoon, Ji Woong (Jay) Han, Hyunsuk Shim
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Novel Inhibitors of NOX2/ROS
Applications
Novel therapeutics for treatment of various inflammatory airway conditions including chronic obstructive pulmonary disease (COPD).
Key Benefits
NOX2/ROS inhibitors have potential for use in a wide variety of clinical indications.
Current treatments are weakly effective or provide only symptomatic relief for inflammatory airway diseases.
Technical...
Published: 7/30/2024
Contributor(s): Thota Ganesh, John David Lambeth, Susan Smith, Aiming Sun
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Functional Restoration of Diabetic Bone Marrow-Derived Cells
Application
Methods for restoration of diabetic bone marrow cell function for cell based therapy, using pre-existing molecules and their derivatives.
Key Benefits
Ability to "reactivate" diabetic bone marrow-derived stem or progenitor cells which also include peripheral blood cells such as endothelial progenitor cells.
Cells are more...
Published: 11/20/2024
Contributor(s): Young-Sup Yoon, Ji Woong (Jay) Han
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Normalization Method Simplifies Inter-Individual Comparisons
Application
Normalization procedure that allows for the direct comparison of high-throughput liquid chromatography/mass spectrometry (LC/MS) metabolomic data gathered from different individuals at different times.
Key Benefits
Samples used can be collected at different times and/or analyzed by different mass spectrometers.
Cumulative datasets...
Published: 11/20/2024
Contributor(s): Dean Jones, Quinlyn Soltow, Jennifer Johnson
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Growth Factor as a Treatment for Obesity
Applications
Therapeutic use of Glial Cell Line-Derived Neurotrophic Factor (GDNF) to treat obesity.
Potential drug target for further therapeutic development.
Key Benefits
Growth Factor expression in the brain causes a reduction in high fat diet-induced obesity.
Fat level reduction has been shown in both mouse and human cells.
Reducing...
Published: 11/20/2024
Contributor(s): Shanthi Srinivasan
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Food Lists for Nutritional Management of Metabolic Disorders
Applications
Booklets which provide a reference guide for nutritional management of rare genetic diseases.
Technical Summary
A majority of inherited metabolic disorders affect children and adults worldwide. Many metabolic disorders require special dietary therapy and monitoring by a team of physicians, nurses, genetic counsellors, social workers,...
Published: 11/20/2024
Contributor(s): Rani Singh
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Neurotrophic Factor as Therapeutic for Diabetes
Application
The use of Glial Cell-derived Neurotrophic Factor (GDNF) or its mimics as a potential therapeutic for diabetes mellitus type 1 (IDDM).
Key Benefits
GDNF enhances pancreatic β-cell growth and survival in vitro and in vivo.
GDNF transgenic mice have lower blood glucose levels and are more resistant to developing diabetes compared...
Published: 7/30/2024
Contributor(s): Shanthi Srinivasan
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