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Gene and Cell therapy
Organoids provide an unprecedented opportunity to study human organ development and physiology, and investigate the cause of disease.
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Genetics
Immunology
Complement 5a increases inflammation in Hunter Syndrome
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Pulmonology
Repurposing a phase I/II compound (AURKB inhibitor - barasertib) for idiopathic pulmonary fibrosis therapy.
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Immunology
Human Natural Killer cells expressing chimeric antigen receptor that targets aberrant immune system signaling that is involved in auto-antibody production associated with Systemic Lupus Erythematosus.
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Neurodegenerative Diseases
Restore GCase activity in the brain for Gaucher and Parkinson's
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Neurology
A new imaging technology to localize and visualize electrical and/or magnetic signals in two or more frequency ranges.
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Neurology
HDAC3 inhibitors enhance myelin repair in white matter injury and demyelinating diseases
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Pulmonology
Novel serum protein biomarkers and functional data analysis-based algorithm used to develop a predictive test for lung function decline.
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Pulmonology
Scientists have shown that src kinase inhibitors are effective at treating CF patients with a frame deletion of F508 in the CFTR gene.
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Asthma
Cysteamine, an FDA approved treatment for cystinosis, has been shown to be an effective oral treatment for steroid resistant asthma including airway hyperresponsiveness (AHR) and airway inflammation.