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AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsStanley N. Cohen, Ning Deng
Estimating the term…
Aspects of the present disclosure include methods of reducing the deleterious impact of a target gene in a cell, such as the deleterious activity of a mutant extended nucleotide repeat (NR) containing target gene in a cell, by contacting the cell with an effective amount of an enantiomeric tetrahydrocarbazolamine compound. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended NR containing target gene may be reduced, e.g., by reducing (and in some instances differentially, including selectively, reducing) the production or activity of toxic expression products (e.g., RNA or protein) encoded by the target gene. Kits and compositions for practicing the subject methods are also provided.
A compound, having the structure:
A method of treating a subject for a disease or condition associated with the deleterious impact of a mutant extended nucleotide repeat containing target gene, the method comprising:
A method of reducing the deleterious impact of a target gene in a cell, the method comprising:
A kit, comprising:
Dependencies are inferred from the claim text.