Una oligomers having reduced off-target effects in gene silencing

a technology of una oligomers and gene silencing, applied in the field of gene silencing techniques, can solve the problems of difficult to carry out, rna interference is the occurrence of off-target effects, and the silencing technique is difficult to achieve, so as to reduce ttr and off-target effects
US20150307881A1Inactive Publication Date: 2015-10-29ARCTURUS THERAPEUTICS
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
ARCTURUS THERAPEUTICS
Publication Date
2015-10-29
Estimated Expiration
Not applicable · inactive patent

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Abstract

This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.
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Description

SEQUENCE LISTING

[0001] This application includes a Sequence Listing submitted electronically herewith as an ASCII file created on Mar. 24, 2015, named ARC1246WO_SL.txt, which is 30,972 bytes in size, and is hereby incorporated by reference in its entirety.BACKGROUND OF THE INVENTION

[0002] A major drawback of gene silencing techniques such as RNA interference is the occurrence of off-target effects. Off-target effects occur when a gene-silencing agent has an effect on a gene product to which it is not targeted, and is therefore unwanted. Off-target effects loom as one of the main hurdles for developing gene silencing therapeutics.

[0003] One method to reduce off-target effects is to intelligently design the structure of the gene silencing agent to avoid effects on genes other than the desired target. In some cases, the gene silencing agents could be pooled so that the concentration of agents having a particular off-target effect would be reduced. In other cases, the gene silencing agent ...

Claims

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