US2010298425A1PendingUtilityA1

Use of Tocotrienols for Elevating IKBKAP Gene Expression and Treating Familial Dysautonomia

Individually held — no corporate assignee on recordPriority: May 14, 2004Filed: Jul 7, 2010Published: Nov 25, 2010
Est. expiryMay 14, 2024(expired)· nominal 20-yr term from priority
A61K 31/355A61P 25/00
41
PatentIndex Score
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Claims

Abstract

The present invention provides methods for elevating IKBKAP gene expression and the level of functional IKAP protein in cells, which are beneficial to human individual, such as an individual suffering from Familial Dysautonomia, by providing one or more tocotrienols alone or in combination with one or more tocopherols to the cells. The present invention also provides methods for treating Familial Dysautonomia by providing tocotrienols alone or in combination with one or more tocopherols to a patient having Familial Dysautonomia. Related therapeutic kits are also provided.

Claims

exact text as granted — not AI-modified
1 . A method for elevating the level of IKBKAP gene expression in a cell comprising contacting the cell with an effective amount of a tocotrienol. 
     
     
         2 . A method of elevating the level of the IKAP protein in a cell comprising contacting the cell with an effective amount of a tocotrienol. 
     
     
         3 . The method of  claim 1 , wherein said cell is derived from an individual or within an individual. 
     
     
         4 . The method of  claim 3 , wherein said individual has Familial Dysautonomia. 
     
     
         5 . The method of  claim 1 , wherein said tocotrienol is selected from the group consisting of α, β, γ, or δ tocotrienol, or a combination thereof. 
     
     
         6 . The method of  claim 1 , wherein the cell is brought into contact in culture with said tocotrienol at a concentration in the range of 0.25-50 μg/ml. 
     
     
         7 . The method of  claim 1 , further comprising contacting the cell with an effective amount of a tocopherol. 
     
     
         8 . The method of  claim 7 , wherein said tocopherol is selected from the group consisting of α, β, γ, or δ tocopherol, or a combination thereof. 
     
     
         9 . The method of  claim 7 , wherein said effective amount of said tocopherol is in the range of 0.25 μg/ml to 50 μg/ml. 
     
     
         10 . A method of elevating the level of IKBKAP gene expression in an individual comprising providing an effective amount of a tocotrienol to said individual. 
     
     
         11 . A method of elevating the level of the IKAP protein in an individual comprising providing an effective amount of a tocotrienol to said individual. 
     
     
         12 . The method of  claim 10 , wherein said individual has Familial Dysautonomia. 
     
     
         13 . The method of  claim 10 , wherein said tocotrienol is provided to said individual at 1 to 1000 mg per day. 
     
     
         14 . The method of  claim 10 , further comprising providing an effective amount of a tocopherol to said individual. 
     
     
         15 . The method of  claim 14 , wherein said tocopherol is selected from the group consisting of α, β, γ, or δ tocopherol, or a combination thereof. 
     
     
         16 . The method of  claim 14 , wherein said tocopherol is provided to said individual at 1 to 1000 mg per day. 
     
     
         17 - 24 . (canceled) 
     
     
         25 . A kit for elevating the level of IKBKAP gene expression or the IKAP protein, comprising an effective amount of a tocotrienol and instructions of use. 
     
     
         26 . The kit of  claim 25 , further comprising an effective amount of a tocopherol. 
     
     
         27 . A method for treating an individual comprising providing an effective amount of a tocotrienol to said individual. 
     
     
         28 . The method of  claim 27 , wherein said tocotrienol is selected from the group consisting of α, β, γ, or δ tocotrienol, or a combination thereof. 
     
     
         29 . The method of  claim 27 , wherein said individual is of any age, from in utero throughout life. 
     
     
         30 . The method of  claim 27 , wherein said tocotrienol is provided to said individual at 25 to 500 mg per day. 
     
     
         31 . The method of  claim 27 , wherein said tocotrienol is provided to said individual orally, enterally or parenterally. 
     
     
         32 . The method of  claim 27 , further comprising providing an effective amount of a tocopherol to said individual. 
     
     
         33 . The method of  claim 32 , wherein said tocopherol is selected from the group consisting of α, β, γ, or δ tocopherol, or a combination thereof. 
     
     
         34 . The method of  claim 32 , wherein said tocopherol is provided to said individual at 1 to 1000 mg per day. 
     
     
         35 . The method of  claim 2 , wherein said cell is derived from an individual or within an individual. 
     
     
         36 . The method of  claim 35 , wherein said individual has Familial Dysautonomia. 
     
     
         37 . The method of  claim 2 , wherein said tocotrienol is selected from the group consisting of α, β, γ, or δ tocotrienol, or a combination thereof. 
     
     
         38 . The method of  claim 2 , wherein the cell is brought into contact in culture with said tocotrienol at a concentration in the range of 0.25-50 μg/ml. 
     
     
         39 . The method of  claim 2 , further comprising contacting the cell with an effective amount of a tocopherol. 
     
     
         40 . The method of  claim 39 , wherein said tocopherol is selected from the group consisting of α, β, γ, or δ tocopherol, or a combination thereof. 
     
     
         41 . The method of  claim 39 , wherein said effective amount of said tocopherol is in the range of 0.25 μg/ml to 50 μg/ml. 
     
     
         42 . The method of  claim 11 , wherein said individual has Familial Dysautonomia. 
     
     
         43 . The method of  claim 11 , wherein said tocotrienol is provided to said individual at 1 to 1000 mg per day. 
     
     
         44 . The method of  claim 11 , further comprising providing an effective amount of a tocopherol to said individual. 
     
     
         45 . The method of  claim 44 , wherein said tocopherol is selected from the group consisting of α, β, γ, or δ tocopherol, or a combination thereof. 
     
     
         46 . The method of  claim 44 , wherein said tocopherol is provided to said individual at 1 to 1000 mg per day.

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