Pak modulators
AssigneeMassachusetts Institute of Technology
InventorsSusumu Tonegawa, Mansuo L. Hayashi, Bridget Dolan
Life of this patent
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Abstract
The present invention provides methods for treating fragile X syndrome and/or other neurodevelopmental disorders by administering p21-activated kinase (PAK) modulators to a patient suffering from, susceptible to, and/or exhibiting one or more symptoms of FXS and/or other neurodevelopmental disorders. The present invention provides PAK modulators and pharmaceutical compositions comprising PAK modulators. The present invention further provides methods for identifying and/or characterizing PAK modulators.
Claims41
A method comprising steps of:
- providing a subject susceptible to, suffering from, or exhibiting at least one symptom associated with fragile X syndrome (FXS), mental retardation, autism spectrum disorders, premature ovarian failure, or fragile X associated tremor ataxia;
- and
- administering an amount of at least one inhibitor of p21-activated kinase (PAK) to the subject effective to treat, alleviate, ameliorate, relieve, delay onset of, inhibit progression of, reduce severity of, or reduce incidence of the at least one symptom associated with FXS.
2-5. (canceled)
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10-15. (canceled)
18-28. (canceled)
30-50. (canceled)
53-55. (canceled)
57-63. (canceled)
66-72. (canceled)
A method comprising steps of:
- providing an FMR knockout mouse exhibiting at least one symptom of fragile X syndrome (FXS);
- administering at least one candidate substance to the mouse;
- and
- measuring the effect of the at least one candidate substance on the at least one symptom of FXS.
75-79. (canceled)
81-86. (canceled)
88-92. (canceled)
94-98. (canceled)
A method comprising steps of:
- providing an FMR knockout mouse exhibiting at least one symptom of mental retardation or autism spectrum disorders;
- administering at least one candidate substance to the mouse;
- and
- measuring the effect of the at least one candidate substance on the at least one symptom of mental retardation or autism spectrum disorders.
A method comprising steps of:
- providing p21-activated kinase (PAK);
- providing a natural binding partner of PAK;
- administering at least one candidate substance to PAK and the natural binding partner;
- and
- measuring the effect of the at least one candidate substance on the binding interaction between PAK and the natural binding partner.
101-108. (canceled)
A method comprising steps of:
- providing a p21-activated kinase (PAK);
- providing a phosphorylation substrate of PAK;
- administering at least one candidate substance to PAK and the phosphorylation substrate of PAK;
- and
- measuring the effect of the at least one candidate substance on ability of PAK to phosphorylate the substrate.
110-128. (canceled)
A pharmaceutical composition comprising:
- a modulator of p21-activated kinase (PAK);
- and
- at least one pharmaceutically acceptable excipient.
(canceled)
A kit comprising:
- an FMR KO mouse;
- a least one candidate substance;
- a positive control, wherein the positive control comprises a known modulator of p21-activated kinase (PAK);
- a negative control, wherein the negative control comprises a substance known to not be a PAK modulator;
- instructions for administering the candidate substance to the mouse in order to determine whether the candidate substance treats, alleviates, ameliorates, relieves, delays onset of, inhibits progression of, reduces severity of, or reduces incidence of one or more symptoms or features of FXS.
Dependencies are inferred from the claim text.