US2015051288A1PendingUtilityA1

Methods and Compositions for Treatment of Autism

Assignee: UNIV JOHNS HOPKINSPriority: Oct 31, 2011Filed: Oct 10, 2014Published: Feb 19, 2015
Est. expiryOct 31, 2031(~5.3 yrs left)· nominal 20-yr term from priority
A61P 25/28G01N 2500/10G01N 33/5023G01N 33/5041G01N 2800/52A61K 31/517A61K 31/18G01N 2800/2814A61K 31/145A61K 31/70A61K 31/343G01N 33/5044A61K 31/167A61K 31/165A61K 31/506A61K 31/19A61K 31/4045A61K 31/4184A61K 31/17A61K 31/26A61K 31/192A61K 36/31A23L 33/115
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Claims

Abstract

Disclosed herein are methods and compositions for treating autism. Disclosed herein are methods and compositions for treating an autism spectrum disorder.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A method of treating autism or one or more autism spectrum disorders, comprising,
 administering to a subject diagnosed with autism or one or more autism spectrum disorders an effective amount of a composition comprising hydroxyurea; and   allowing the cells of the subject to return to homeostasis substantially equivalent to the state that existed in the cells prior to administering the compound.   
     
     
         2 . The method of  claim 1 , wherein in at least one cell of the subject, the stress proteome is stimulated. 
     
     
         3 . The method of  claim 1 , wherein in at least one cell of the subject, increased nitrous oxide production is detected. 
     
     
         4 . The method of  claim 1 , wherein in at least one cell of the subject stress-sensing organelles are increased from an amount prior to the administration of the composition. 
     
     
         5 . The method of  claim 4 , wherein the stress-sensing organelle is a mitochondrion. 
     
     
         6 . The method of  claim 4 , wherein the stress-sensing organelle is a peroxisome. 
     
     
         7 . The method of  claim 1 , wherein the general cellular stress response comprises at least one of mitochondrial biogenesis, peroxisome proliferation, activation of the stress proteome, transcription and/or translation of genes and proteins encoded by genes comprising heat shock and unfolded protein, genes for autophagic responses, genes for antioxidant responses, and genes for the c-jun-N-terminal kinase pathway. 
     
     
         8 . The method of  claim 7 , wherein genes for heat shock proteins comprise heat shock protein 40, 70, and/or 90 family members; unfolded protein genes comprise glucose regulated protein 78 (BIP), PERK, inositol requiring 1 (IRE1) and activating transcription factor 6; autophagic response genes comprise beclin-1 (BCN1), autophagy protein 5 (ATG5) and microtubule-associated protein 1 light chain 3 (LC3 or APG8); and antioxidant response genes comprises expression of nuclear factor erythroid 2-like 2 (NFE2L2), heme oxygenase 1 (HMOX1) and superoxide dismutase 2 (SOD2). 
     
     
         9 . The method of  claim 1 , wherein the at least one cell of the subject is located in the brain of the subject. 
     
     
         10 . The method of  claim 1 , wherein the at least one cell of the subject is not located in the brain of the subject. 
     
     
         11 . The method of  claim 1 , wherein the composition comprises at least one of a histone deacetylase inhibitor, Class I histone deacetylase inhibitor, or a Class II histone deacetylase inhibitor. 
     
     
         12 . The method of  claim 1 , wherein the composition is a pharmaceutical composition, a medical food, a food, a drink, or a dietary supplement. 
     
     
         13 . A method of treating autism or one or more autism spectrum disorders, comprising, administering to a subject diagnosed with autism or one or more autism spectrum disorders an effective amount of a pharmaceutical composition comprising hydroxyurea. 
     
     
         14 . A method for determining effectiveness of a compound in treating autism or one or more autism related disorders, comprising,
 contacting a first cell with an effective amount of a test compound,   comparing the response of the first cell to the response of a second, optionally identical, cell that was contacted with hydroxyurea, and   determining whether or not the test compound induces a general cellular stress response in cells.   
     
     
         15 . The method of  claim 14 , wherein the cell is a normal human fibroblast, a XALD fibroblast, or a K562 cell. 
     
     
         16 . The method of  claim 14 , wherein the contacting occurs in vitro or in vivo. 
     
     
         17 . A method of treating autism or one or more autism spectrum disorders, comprising,
 administering to a subject diagnosed with autism or one or more autism spectrum disorders an effective amount of a composition comprising hydroxy that induces a general cellular stress response in at least one cell of the subject; and   allowing the cells of the subject to return to homeostasis substantially equivalent to the state that existed in the cells prior to administering the compound.

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