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US 2020/0400651 A1Published application
Method of using human spheroids for drug discovery
AssigneeStemoniX Inc.
InventorCassiano Carromeu
Life of this patent
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Abstract
The present invention discloses, in one embodiment, a method of using human induced pluripotent stem cells to generate three-dimensional human organ tissue for therapeutic drug toxicity and discovery. In one embodiment, a high throughput microtiter plate is loaded with both wild type and Rett disease 3D spheroids and exposed to a drug library, and activity is measured and analyzed for disease rescue to wild type cell behavior.
Claims20
A method to detect the effect of one or more compounds on spheroids, comprising:
- contacting one or more spheroids of human cells of uniform diameter and one or more test compounds, wherein the spheroids are obtained from cells of an autism patient, cells of a Rett syndrome patient, or cells that are from a model of Rett syndrome;
- and
- detecting the effect of the one or more compounds on one or more of the spheroids.
A method or prevent, inhibit or treat one or more symptoms of Rett syndrome in a human, comprising:
- administering to the patient a composition comprising an effective amount of one or more of a 5-HT4 receptor selective agonist, a zabicycloalkyl benzimidazolone, a GABA receptor antagonist, a benzodiazepine, a competitive antagonist at the benzodiazepine receptor, an acetazolamide, a selective noradrenaline reuptake inhibitor, an antimuscarinic, a selective serotonin receptor agonist, a compound that enhances release of gonadotropin releasing hormone, a selective serotonin reuptake inhibitor, a branched-chain saturated fatty acid anion, an inhibitor of CYP2C9, an inhibitor of glucuronyl transferase, an inhibitor of histone deacetylase, an inhibitor of epoxide hydrolase, a gamma-aminobutyric acid (GABA) agonist, a nonsteroidal anti-inflammatory drug (NSAID), a biindole, an anticholinergic, a dopamine antagonist, an acetylcholinesterase inhibitor, an antihistamine, a benzothiadiazine, a modulator of glutamate AMPA receptors, an anti-emetic, a serotonin inverse agonist, an inhibitor of monoamine oxidase, or an alkaloid, or any combination thereof.
Dependencies are inferred from the claim text.