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Compositions and methods for modulation of SMN2 splicing in a subject
US8980853B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-8980853-B2 |
| Application number | US-201013380021-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jun 17, 2010 |
| Priority date | Jun 17, 2009 |
| Publication date | Mar 17, 2015 |
| Grant date | Mar 17, 2015 |
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Abstract
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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.
First claim
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What is claimed is: 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.
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Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
for peripheral neuropathies · CPC title
Drugs for disorders of the nervous system · CPC title
for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia · CPC title
2'-O-R Modification · CPC title
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- What does patent US8980853B2 cover?
- 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.
- Who is the assignee on this patent?
- Bennett C Frank, Hung Gene, Rigo Frank, and 8 more
- What technology area does this patent fall under?
- Primary CPC classification A61K31/7088. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Mar 17 2015 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).