Gene therapy for duchenne muscular dystrophy
US-2024390522-A1 · Nov 28, 2024 · US
US9303078B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9303078-B2 |
| Application number | US-201013500612-A |
| Country | US |
| Kind code | B2 |
| Filing date | Oct 8, 2010 |
| Priority date | Oct 8, 2009 |
| Publication date | Apr 5, 2016 |
| Grant date | Apr 5, 2016 |
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The present invention provided methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated simple or double trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and to mediate a simple or double trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (Chimeric RNA).
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The invention claimed is: 1. A nucleic acid molecule comprising: a) two target binding domains AS and AS′ that target the binding of the nucleic acid molecule to a target pre-mRNA, wherein said two target binding domains AS and AS′ are located respectively at the 5′-end and at the 3′-end of the nucleic acid molecule, b) a 3′ splice region comprising a branch point, a polypyrimidine tract and a 3′ splice acceptor site, c) a 5′ splice region comprising a 5′ splice donor site,…
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