Methods for treating familial dysautonomia
OwnerBerish Rubin, Sylvia Andersoninventors
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Abstract
The present invention provides methods for modulating mRNA splicing in a normal or diseased cell or individual, e.g., elevating wild-type IKBKAP transcripts and the level of functional IKAP protein in an individual suffering from Familial Dysautonomia, by providing catechins, such as EGCG, to the cell or individual. The present invention also provides methods for treating Familial Dysautonomia by providing EGCG-related catechins to an individual having Familial Dysautonomia. Related therapeutic kits are also provided.
Claims19
A method for modulating the level of an alternatively spliced mRNA transcript in a cell comprising contacting the cell with an effective amount of at least one catechin.
A method of elevating the level of the IKAP protein in a cell comprising contacting the cell with an effective amount of at least one EGCG-related catechin.
A method of elevating the level of the wild-type IKBKAP transcript in an individual comprising providing an effective amount of at least one catechin to said individual.
A method of elevating the level of the IKAP protein in an individual comprising providing an effective amount of at least one catechin to said individual.
A method for treating an FD individual comprising providing an effective amount of at least one catechin to said individual.
A kit for treating an individual having FD, comprising an effective amount of at least one catechin and instructions for use.
Dependencies are inferred from the claim text.