Antisense oligonucleotides directed to huntingtin
OwnerGene Hung, C. Frank Bennett +2inventors
InventorsGene Hung, C. Frank Bennett, Janet M. Leeds, Susan M. Freier
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Abstract
Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.
Claims13
An antisense oligonucleotide 12 to 35 nucleotides in length comprising:
- (i) at least 13 consecutive nucleotides of SEQ ID NO:104, having at least one modified internucleoside linkage, sugar moiety, or nucleobase;
- and
- (ii) a chimeric oligonucleotide having a gap segment positioned between 5′ and 3′ wing segments.
Dependencies are inferred from the claim text.