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:
Jan. 21, 2014

Filed:

May. 27, 2009
Applicants:

Sergey V. Paushkin, Belle Mead, NJ (US);

Nikolai Naryshkin, East Brunswick, NJ (US);

Charles Romfo, Easton, PA (US);

Ellen Welch, Califon, NJ (US);

Inventors:

Sergey V. Paushkin, Belle Mead, NJ (US);

Nikolai Naryshkin, East Brunswick, NJ (US);

Charles Romfo, Easton, PA (US);

Ellen Welch, Califon, NJ (US);

Assignee:

PTC Therapeutics, Inc., South Plainfield, NJ (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12Q 1/68 (2006.01); A01N 43/00 (2006.01); C12N 15/00 (2006.01);
U.S. Cl.
CPC ...
Abstract

The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.


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