MODIFIED DOUBLE-STRANDED RNA AGENTS

One aspect of the present disclosure relates to a dsRNA agent comprising a sense strand and an antisense strand sufficiently complementary to at least one portion of a mRNA of the target gene, each strand having 14 to 40 nucleotides, wherein the antisense strand comprises at least one TNA (threose n...

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Bibliographic Details
Main Authors MATSUDA, Shigeo, MANOHARAN, Muthiah, DATTA, Dhrubajyoti, MIKAMI, Atsushi
Format Patent
LanguageEnglish
French
German
Published 06.08.2025
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
DERIVATIVES THEREOF
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
SUGARS
VINEGAR
WINE
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Summary:One aspect of the present disclosure relates to a dsRNA agent comprising a sense strand and an antisense strand sufficiently complementary to at least one portion of a mRNA of the target gene, each strand having 14 to 40 nucleotides, wherein the antisense strand comprises at least one TNA (threose nucleic acid) at position 1, counting from the 5' end. Other aspects of the invention relate to a method of modulating the expression of a target gene in a cell and a method of treating a subject having a CNS disorder comprising administering to a cell or a subject the dsRNA agent.
Bibliography:Application Number: EP20230800673