Nucleic acid-controlled catalytic rnas for trigger-responsive regulation
US-2024425855-A1 · Dec 26, 2024 · US
US9012425B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9012425-B2 |
| Application number | US-201214125063-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jun 8, 2012 |
| Priority date | Jun 10, 2011 |
| Publication date | Apr 21, 2015 |
| Grant date | Apr 21, 2015 |
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The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.
Opening claim text (preview).
The invention claimed is: 1. A method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene comprising administering to the patient an effective amount of an antisense oligonucleotide which is SEQ ID NO:2 (ESE(+50+70)).
Human Necessities · mapped topic
Chemistry & Metallurgy · mapped topic
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