Treatment of rett syndrome and other disorders

Abstract

The invention relates to methods for treatment of Rett Syndrome and other disorders of synaptic function and maturation using IGF1, (1-3)IGF-1, (1-3)IGF-1 analog(s) and/or related therapeutic molecules.

1 .- 21 . (canceled) 
     
     
         22 . A method for treating a disorder of synaptic function and/or maturation in a subject, comprising
 administering to a subject in need of such treatment an effective amount of insulin-like growth factor 1 (IGF1) and/or (1-3)IGF 1 to treat the subject.   
     
     
         23 . The method of  claim 22 , wherein IGF1 is administered. 
     
     
         24 . The method of  claim 23 , wherein the IGF1 is recombinant IGF1. 
     
     
         25 . The method of  claim 23 , wherein the IGF1 is human IGF1. 
     
     
         26 . The method of  claim 23 , wherein the dose of IGF1 administered is about 0.1-10 mg/kg/day. 
     
     
         27 . The method of  claim 26 , wherein the dose of IGF1 administered is about 0.1-2 mg/kg/day. 
     
     
         28 . The method of  claim 22 , wherein (1-3)IGF-1 is administered. 
     
     
         29 . The method of  claim 28 , wherein the dose of (1-3)IGF-1 administered is about 0.1-100 mg/kg/day. 
     
     
         30 . The method of  claim 29 , wherein the dose of (1-3)IGF-1 administered is about 6-20 mg/kg/day. 
     
     
         31 .- 34 . (canceled) 
     
     
         35 . The method of  claim 22 , wherein the subject is a human. 
     
     
         36 . The method of  claim 22 , wherein the IGF1 and/or (1-3)IGF1 is administered orally, intravenously, intramuscularly, intranasally, intraperitoneally, subcutaneously, or intrathecally. 
     
     
         37 . The method of  claim 22 , wherein the IGF1 and/or (1-3)IGF1 is administered after diagnosis of the disorder. 
     
     
         38 . The method of  claim 22 , wherein the IGF1 and/or (1-3)IGF1 is administered prophylactically before diagnosis of the disorder. 
     
     
         39 . The method of  claim 22 , wherein the disorder is autism, autism spectrum disorder, Angelmann's Syndrome, tuberous sclerosis, Fragile X syndrome, schizophrenia, depression, neurodegenerative disorders including Parkinson's disease, Huntington's disease and Alzheimer's disease, stroke or trauma. 
     
     
         40 . The method of  claim 22 , wherein the subject is free of symptoms otherwise calling for treatment with the IGF1 and/or (1-3)IGF1. 
     
     
         41 . The method of  claim 22 , further comprising first testing the subject for a mutation in a gene that is a genetic basis for the disorder or a gene that is a target of or downstream of such a gene. 
     
     
         42 . The method of  claim 22 , further comprising administering to the subject a second therapeutic, wherein the second therapeutic is tPA, BDNF, a molecule that regulates inhibition such as a benzodiazepine, or a molecule that is a neurotransmitter agonist, antagonist or analog, and wherein the second therapeutic and the IGF1 and/or (1-3)IGF-1, are administered in a combined amount effective to treat the subject. 
     
     
         43 . The method of  claim 22 , wherein the amount of IGF1 and/or (1-3)IGF1 is effective to restore synaptic function and/or maturation, consolidate synapses and/or regulate neuronal plasticity. 
     
     
         44 . A method for increasing synaptic maturation comprising
 contacting one or more neurons comprising one or more synapses with an amount of insulin-like growth factor 1 (IGF1) and/or (1-3)IGF-1 effective to increase the maturation of the one or more synapses of the one or more neurons.   
     
     
         45 .- 57 . (canceled) 
     
     
         58 . The method of  claim 22 , wherein the IGF1 and/or (1-3)IGF-1 is administered as a pharmaceutical composition. 
     
     
         59 . The method of  claim 58 , wherein the pharmaceutical composition comprises IGF1 and/or (1-3)IGF-1 combined with a stabilizing agent or carrier. 
     
     
         60 . The method of  claim 59 , wherein the stabilizing agent or carrier is a polyethylene glycol.