Sarepta Therapeutics, Inc.

Avi Biopharma, Inc.

Bao Zhong Cai

Dwight D Weller

Gunnar J Hanson

Richard Keith Bestwick

Ming Zhou

Alexander Charles Rudolph

Jed N Hassinger

Mitchell Martini

Ross Shimabuku

Katie Thomas

Diane Elizabeth Frank

Charles Rudolph

Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5&#x2032;-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.

Functionally-modified oligonucleotides and subunits thereof

Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5&#x2032;-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.

Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups

Oligonucleotide analogs comprising modified intersubunit linkages and/or modified 3' and/or 5&#x2032;-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.

Functionally-modified oligonucleotide analogs comprising modified intersubunit linkages and/or modified 3' and/or 5&#x2032;-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.

Morpholino oligomers containing both uncharged and cationic intersubunit linkages are provided. The oligomers are oligonucleotide analogs containing predetermined sequences of base-pairing moieties. The presence of the cationic intersubunit linkages in the oligomers, typically at a level of about 10-50% of total linkages, provides enhanced antisense activity, in various antisense applications, relative to the corresponding uncharged oligomers. Also provided are such oligomers conjugated to peptide transporter moieties, where the transporters are preferably composed of arginine subunits, or arginine dimers, alternating with neutral amino acid subunits.

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Corvallis, OR, United States of America

Bao Zhong Cai