DNA-ZYME BASED METHODS & COMPOSITIONS FOR TREATING HUNTINGTON'S DISEASE
Provided herein are methods and compositions for treating Huntington's disease. For example, provided are compositions that include DNA oligonucleotides that have targeting specificity for RNA encoding mutant huntingtin protein. When introduced into a cell, the DNA oligonucleotides reduce expression of the mutant huntingtin protein in a cell. For example, the DNA oligonucleotide, functioning as an enzymatic DNA molecule (or DNAzyme), cleaves the RNA encoding the mutant mRNA protein, thereby rendering the mRNA incapable of expression. That is, the cleavage event renders the RNA non¬ functional and reduces or abrogates protein expression from that RNA. Hence, synthesis of the mutant huntingtin protein is selectively reduced or inhibited, in accordance with the methods and compositions described herein. By silencing or reducing the expression of the mutant huntingtin protein, the methods and compositions describe herein can be used to treat Huntington's disease, including adult and juvenile onset Huntington's disease.