Rna interference for the treatment of gain-of-function disorders
AssigneeUniversity of Massachusetts
InventorsNeil Aronin, Phillip D. Zamore
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Abstract
The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
Claims15
1-51. (canceled)
A composition for treating a subject having or at risk for a disease characterized or caused by a gain-of-function mutant protein, comprising:
- a vector that expresses an effective amount of an RNAi agent targeting an allelic polymorphism within a gene encoding said mutant protein.
A composition for treating a subject having or at risk for Huntington's disease characterized or caused by a mutant huntingtin (htt) protein, comprising a vector that expresses an effective amount of a siRNA targeting a heterozygous single nucleotide polymorphism (SNP) within a gene encoding the mutant htt protein, such that RNA silencing of said gene occurs, wherein the siRNA comprises (i) an antisense strand or a variant thereof having sufficient complementarity to the SNP at genomic site RS362307 to direct target-specific RNA interference of the gene encoding the mutant htt protein;
- and (ii) a sense strand or a variant thereof that is substantially complementary to the antisense strand, such that the sense and antisense strands are capable of annealing together.
A composition of treating a subject having or at risk for Huntington's disease characterized or caused by a mutant huntingtin (htt) protein, comprising a vector that expresses an effective amount of a siRNA targeting a heterozygous single nucleotide polymorphism (SNP) within a gene encoding the mutant htt protein, such that RNA silencing of said gene occurs, wherein the siRNA comprises (i) an antisense strand or a variant thereof having sufficient complementarity to the SNP at genomic site RS362331 or RS363125 to direct target-specific RNA interference of the gene encoding the mutant htt protein;
- and (ii) a sense strand or a variant thereof that is substantially complementary to the antisense strand, such that the sense and antisense strands are capable of annealing together.
Dependencies are inferred from the claim text.