Anti-C9ORF72 oligonucleotides and related methods
AssigneeUniversity of Massachusetts
InventorsRobert H. Brown, Jr., Jonathan K. Watts, Helene Tran, Michael Moazami
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Abstract
The present disclosure provides antisense compounds, methods, and compositions for silencing C9ORF72 transcripts. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with C9ORF72 in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with C9ORF72.
Claims33
An antisense oligonucleotide comprising a region of complementarity to a C9ORF72 sense transcript sequence of 5′ GAGUCGCGCGCUAGGGGC 3′ (SEQ ID NO:
- 9), wherein the antisense oligonucleotide comprises internucleotide linkages from 5′ to 3′ of sooosssssssssooos, wherein each s is a phosphorothioate linkage and each o is a phosphodiester linkage.
An antisense oligonucleotide comprising the sequence GsCoCoCoCsTsAsGsCsGsCsGsCsGoAoCoTsC, wherein
- s represents a phosphorothioate internucleotide linkage;
- o represents a phosphodiester internucleotide linkage;
- A is an adenosine comprising a 2′-O-(2-methoxyethyl) modification;
- G is a guanosine comprising a 2′-O-(2-methoxyethyl) modification;
- C is a cytidine comprising a 2′-O-(2-methoxyethyl) modification;
- T is a thymine comprising a 2′-O-(2-methoxyethyl) modification;
- and
- each cytosine is a 5-methylcytosine.
Dependencies are inferred from the claim text.