EXTRAHEPATIC DELIVERY

One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more...

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Bibliographic Details
Main Authors JADHAV, Vasant R, MANOHARAN, Muthiah, ZLATEV, Ivan, PIERSON, Justin M, SALINAS, Juan C, JUNG, Michelle H, LENTINI, Scott P, MATSUDA, Shigeo, MAIER, Martin A, JANAS, Maja, MILSTEIN, Stuart, KEL'IN, Alexander V, NAIR, Jayaprakash K, GUENTHER, Dale C, DATTA, Dhrubajyoti, HE, Guo, THEILE, Christopher S
Format Patent
LanguageEnglish
Published 19.01.2023
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
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Summary:One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic monomer-conjugated compound.
Bibliography:Application Number: US202017774477