The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Nov. 04, 2014

Filed:

Sep. 23, 2011
Applicants:

Patrick L. Iversen, Corvallis, OR (US);

Dwight D. Weller, Corvallis, OR (US);

Jed N. Hassinger, Corvallis, OR (US);

Inventors:

Patrick L. Iversen, Corvallis, OR (US);

Dwight D. Weller, Corvallis, OR (US);

Jed N. Hassinger, Corvallis, OR (US);

Assignee:

Sarepta Therapeutics, Inc., Corvallis, OR (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); C12N 15/11 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); C12N 2320/32 (2013.01); C12N 2310/3513 (2013.01); C12N 15/111 (2013.01); C12N 2310/331 (2013.01); A61K 48/0008 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3233 (2013.01); C12N 15/1135 (2013.01); C12N 2310/3145 (2013.01);
Abstract

A therapeutic oligomer-peptide conjugate, and methods of using the conjugate are disclosed. The conjugate includes (a) a substantially uncharged oligonucleotide analog compound having a base sequence that includes a string of bases that are complementary to four or more contiguous cytosine bases in a target nucleic acid region to which the compound is intended to bind, and (b) conjugated to the compound, an arginine-rich peptide effective to enhance the uptake of the compound into target cells. The string of bases in the compound includes at least one inosine base positioned in the string so as to limit the number of contiguous guanine bases in said string to three or fewer. The conjugate has greater cellular uptake than the compound alone, by virtue of the arginine-rich peptide, and substantially greater antisense activity greater activity than the conjugate in the absence of inosine-for guanine substitutions.


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