Sarepta Therapeutics, Inc.
Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups

Abstract:

Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5'-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.