Nucleic acid-controlled catalytic rnas for trigger-responsive regulation
US-2024425855-A1 · Dec 26, 2024 · US
US10858650B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10858650-B2 |
| Application number | US-201515522171-A |
| Country | US |
| Kind code | B2 |
| Filing date | Oct 30, 2015 |
| Priority date | Oct 30, 2014 |
| Publication date | Dec 8, 2020 |
| Grant date | Dec 8, 2020 |
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Methods and compositions for modulation of the activity of alpha thalassemia/mental retardation syndrome X-linked (ATRX), e.g., modulation of DNA-ATRX or RNA-ATRX interactions, and methods for identifying and using compounds that modulate DNA-ATRX or RNA-ATRX interactions, as well as the compounds themselves.
Opening claim text (preview).
What is claimed is: 1. A method of treating a subject who has Rett Syndrome, the method comprising administering to the subject a therapeutically effective amount of a nucleic acid oligonucleotide that targets a sequence within SEQ ID NOs. 7957, 7959, 7961, or 7962. 2. The method of claim 1 , wherein the nucleic acid oligonucleotide comprises one or more of DNA, RNA, PNA, HNA, MNA, ANA, LNA, CAN, INA, CeNA, TNA, (2′-NH)-TNA, (3′-NH)-TNA, alpha-L-Ribo-LNA, alpha-L-Xylo-LNA, beta-D-Ribo-LNA, beta-D-Xylo-LNA, [3.2.1]-LNA, Bicyclo-DNA, 6-Amino-Bicyclo-DNA, 5-epi-Bicyclo-DNA, alpha-Bicyclo-DNA, Tricyclo-DNA, Bicyclo[4.3.0]-DNA, Bicyclo[3.2.1]-DNA, Bicyclo[4.3.0]amide-DNA, beta-D-Ribopyranosyl-NA, alpha-L-Lyxopyranosyl-NA, 2′-R-RNA, 2′-OR-RNA, 2′-AE-RNA, alpha-L-RNA, and beta-D-RNA.
Tetrahymena · CPC title
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title
Hydrolases (3) · CPC title
interfering nucleic acids [NA] · CPC title
General methods of preparing gene libraries, not provided for in other subgroups · CPC title
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