Claims for Patent: 8,980,853

Summary for Patent: 8,980,853

Title:	Compositions and methods for modulation of SMN2 splicing in a subject
Abstract:	Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
Inventor(s):	Bennett; C. Frank (Carlsbad, CA), Hung; Gene (San Diego, CA), Rigo; Frank (Carlsbad, CA), Krainer; Adrian R. (Huntington Square, NY), Hua; Yimin (Jericho, NY), Passini; Marco A. (Shrewsbury, MA), Shihabuddin; Lamya (Brighton, MA), Cheng; Seng H. (Natick, MA), Klinger; Katherine W. (Sudbury, MA)
Assignee:	Isis Pharmaceuticals, Inc. (Carlsbad, CA) Cold Spring Harbor Laboratory (Cold Spring Harbor, NY)
Application Number:	13/380,021
Patent Claims:	1. A method comprising administering by a bolus injection into the intrathecal space of a subject with infantile-onset type I spinal muscular atrophy (SMA) an antisense compound comprising an antisense oligonucleotide consisting of 18 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence consisting of the nucleobase sequence SEQ ID NO: 1, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage, wherein each nucleoside of the oligonucleotide is a 2'-MOE nucleoside, and wherein the administering of the antisense compound ameliorates at least one symptom of SMA in the subject. 2. The method of claim 1, wherein the antisense compound is administered at a dose from 0.5 to 10 milligrams of antisense compound per kilogram of body weight of the subject. 3. The method of claim 1, wherein inclusion of exon 7 of SMN2 mRNA in a motoneuron in the subject is increased. 4. The method of claim 1, wherein a 5 mg to 20 mg dose of antisense is administered.