[EN] siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS<br/>[FR] ARNSI POSSÉDANT AU MOINS DEUX LIGANDS À DEUX EXTRÉMITÉS DISTINCTES
申请人:SILENCE THERAPEUTICS GMBH
公开号:WO2019193189A1
公开(公告)日:2019-10-10
There is provided inter alia a conjugate for inhibiting expression of a target gene in a cell, said conjugate comprising a nucleic acid portion and ligand portions, said nucleic acid portion comprising at least one duplex region that comprises at least a portion of a first RNA strand and at least a portion of a second RNA strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene, said ligand portions comprising a linker moiety and a targeting ligand for in vivo targeting of cells and being conjugated exclusively to the 3' and/or 5' ends of one or both RNA strands, wherein the 5' end of the first RNA strand is not conjugated, wherein: (i) the second RNA strand is conjugated at the 5' end to the targeting ligand, and wherein (a) the second RNA strand is also conjugated at the 3' end to the targeting ligand and the 3' end of the first RNA strand is not conjugated; or (b) the first RNA strand is conjugated at the 3' end to the targeting ligand and the 3' end of the second RNA strand is not conjugated; or (c) both the second RNA strand and the first RNA strand are also conjugated at the 3' ends to the targeting ligand; or (ii) both the second RNA strand and the first RNA strand are conjugated at the 3' ends to the targeting ligand and the 5' end of the second RNA strand is not conjugated.
[EN] NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF PROS1 IN A CELL<br/>[FR] ACIDES NUCLÉIQUES POUR INHIBER L'EXPRESSION DE PROS1 DANS UNE CELLULE
申请人:UNIV BERN
公开号:WO2020225301A1
公开(公告)日:2020-11-12
The invention relates to nucleic acid products that interfere with PROS1 gene expression or inhibit its expression. The nucleic acids are particularly for use in the treatment, prevention or reduction of risk of suffering from a bleeding disorder.
[EN] NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF CNNM4 IN A CELL<br/>[FR] ACIDES NUCLÉIQUES POUR INHIBER L'EXPRESSION DE CNNM4 DANS UNE CELLULE
申请人:SILENCE THERAPEUTICS GMBH
公开号:WO2021239825A1
公开(公告)日:2021-12-02
The invention relates to nucleic acid products that interfere with or inhibit CNNM4 (Cyclin M4) gene expression. It further relates to therapeutic uses of CNNM4 inhibition for the treatment of diseases, such as liver diseases including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), liver steatosis, liver fibrosis, liver cirrhosis, liver cancer and other diseases associated with magnesium dysregulation.
[EN] siRNAs WITH VINYLPHOSPHONATE AT THE 5' END OF THE ANTISENSE STRAND<br/>[FR] SIARNS AVEC VINYLPHOSPHONATE À L'EXTRÉMITÉ 5' DU BRIN ANTISENS
申请人:SILENCE THERAPEUTICS GMBH
公开号:WO2019193144A1
公开(公告)日:2019-10-10
The present invention relates to nucleic acids for inhibiting expression of a target gene in a cell, comprising at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene to be inhibited. The first strand of the nucleic acid has a terminal 5' (E)-vinylphosphonate nucleotide that is linked to the second nucleotide in the first strand by a phosphodiester linkage.
(E)-α-Sulfonamidocrotylboronates as Reagents for the Stereoselective Homoaldol Synthesis