Salts and solid forms of (S)-3-(4- (4-(morpholinomethyl)benzyl)oxy)-1-oxoisoindolin-2-yl)piperidine-2,6-dione and compositions comprising and methods of using the same
US-9221788-B2 · Dec 29, 2015 · US
US8986935B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-8986935-B2 |
| Application number | US-200913058653-A |
| Country | US |
| Kind code | B2 |
| Filing date | Aug 13, 2009 |
| Priority date | Aug 13, 2008 |
| Publication date | Mar 24, 2015 |
| Grant date | Mar 24, 2015 |
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Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of the compounds identified by the methods described herein to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Atrophy.
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What is claimed: 1. A method for the identification of a compound that produces a stabilized SMNΔEx7 protein comprising: (A) contacting a compound with either a host cell containing an mRNA transcript transcribed from a nucleic acid construct, or a composition comprising a cell-free extract and an mRNA transcript transcribed from a nucleic acid construct, wherein the nucleic acid construct comprises, in 5′ to 3′ order: (a) a start codon; (b) a fragment of the nucleic acid resid…
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
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