sirna

siRNA Negative Control, a 21-nucleotide siRNA, serves as a negative control.

DLin-KC2-DMA is a cationic/ionizable lipid. Which is used for siRNA delivery.

D-Lin-MC3-DMA, an ionizable cationic lipid, serves as a potent siRNA delivery vehicle.

Enoxacin (NSC-629661) is a broad-spectrum 6-fluoronaphthyridinone antibacterial agent (fluoroquinolones) structurally related to nalidixic acid.

Enoxacin hydrate (AT-2266 hydrate) is a broad-spectrum 6-fluoronaphthyridinone antibacterial agent.

UNC10217938A HCl (UNC7938 HCl) is a 3-deazapteridine analog. UNC10217938A HCl exhibits strong oligonucleotide enhancing effects. UNC10217938A HCl can modulate their intracellular trafficking and release from endosomes, leading to enhance oligonucleotides effects. UNC10217938A HCl also enhances the effects of siRNA and antisense oligonucleotides.

DLinDAP (D-LinDAP) is an ionizable cationic lipid used for SiRNA delivery.

DLinDMA, as a benchmark, is a key stable nucleic acid lipid particleslipid component.

DOPE (DOPE) is a neutral helper lipid for cationic liposomes.It is used in combination with cationic phospholipids to increase efficiency during DNA transfection studies as a non-viral method of gene delivery.

Xalnesiran (sodium) is an siRNA designed for the treatment of chronic hepatitis B (HBV).

Inclisiran (ALN-PCSsc), a double-stranded small interfering RNA (siRNA) compound, serves as the antisense of itself and targets PCSK-9 transcription inhibition. It is employed in the study of hyperlipidemia and cardiovascular disease (CVD) [1].

Mono-Pal-MTO, a palm oil-derived lipid formed by integrating the anticancer agent mitoxantrone (MTO) with palmitoleic acid, exhibits enhanced anticancer activity and efficient siRNA cell delivery when its nanoparticles are mixed with those of di-Pal-MTO at a molar ratio of 1:1 [1].

Inclisiran (unconjugated/naked) is a double-stranded small interfering RNA (siRNA) that lacks GalNAc conjugation. This compound selectively inhibits the transcription of PCSK-9, making it applicable in the research of hyperlipidemia and cardiovascular disease (CVD) [1].

Cemdisiran, an investigational N-acetylgalactosamine-conjugated RNAi (RNA interference) agent with terminal sugar modification, is a small interfering RNA (siRNA) designed to specifically target and suppress the C5 mRNA, thereby reducing the liver's production of the complement component C5 [1].

YSK 05, a pH-sensitive cationic lipid, enhances the intracellular trafficking of non-viral vectors, significantly improves gene silencing and hemolytic activities in its modified form YSK 05-MEND, and effectively overcomes the suppression of endosomal escape caused by PEGylation, thereby enhancing siRNA delivery both in vitro and in vivo [1].

CLinDMA, a cationic lipid, is known to induce an inflammatory response and serves as a precursor in the synthesis of LNP201, a liposome assembly designed for the systemic delivery of siRNA [1].

Lumasiran (ALN-G01), a siRNAproduct, targets glycolate oxidase to inhibit hepatic oxalate production. By silencing the gene encoding glycolate oxidase, Lumasiran depletes the enzyme, preventing the synthesis of oxalate, a toxic metabolite directly linked to the clinical manifestations of Primary hyperoxaluria type 1 (PH1).

DMG-PEG 2000 (DMG-PEG2000) is a lipid excipient that has been used in combination with other lipids in the formation of lipid nanoparticles. Formulations containing DMG-PEG 2000 have been used in the development of lipid nanoparticles for the delivery of mRNA-based vaccines.

C14-SPM, a branched-chain polyamine lipidoid, facilitates the delivery of siRNA [1].

VnP-16, a synthetic peptide fragment mirroring amino acids 270-281 of vitronectin (a multifunctional glycoprotein involved in cell attachment, migration, and spreading), promotes attachment and spreading of isolated human osteogenic cells. This effect is reversible with β1 integrin siRNA knockdown when VnP-16 is applied at 9.1 µg/cm2 on cell plates. The compound enhances osteogenesis in human skin-derived mesenchymal precursors and MC3T3-E1 mouse calvarial osteoblast precursors. Furthermore, in vivo studies show VnP-16 mitigates IL-1α-induced bone loss, reduces calvarial osteoclast numbers in mice, increases bone mineral density, and lessens trabecular bone loss in a mouse model of ovariectomy-induced bone loss.

PF-04523655 is a siRNA targeting the RTP801 gene, an inhibitor of the mammalian target of rampamycin complex 1 (mTORC1) and the downstream transcription factor HIF-1.

Bevasiranib sodium, an siRNA compound, targets and silences the genes responsible for producing vascular endothelial growth factor (VEGF). The suppression of VEGF, a critical factor in the development of choroidal neo-vascularization (CNV), aims to thwart the progression to wet age-related macular degeneration (wet AMD).

Bamosiran, a small interfering RNA (siRNA), specifically targets the β-adrenergic receptor 2 to reduce intraocular pressure.

Inclisiran AS(3n-2), an antisense variant of Inclisiran, features a 2 bp spacer followed by 3 random N sites. Inclisiran itself is a double-stranded small interfering RNA (siRNA) molecule designed to suppress PCSK-9 transcription, thereby reducing LDL cholesterol levels [1].

(Rac)-Z-Phe-Phe-FMK (Cathepsin L-IN-2) is a cathepsin L inhibitor that inhibits the tendency of β-amyloid to induce apoptotic changes .

Patisiran sodium, a double-stranded small interfering RNA (siRNA), selectively targets a specific sequence within the transthyretin (TTR) messenger RNA (mRNA), effectively blocking hepatic synthesis of both mutant and wild-type TTR. It has significant applications in hereditary TTR amyloidosis research.

SST-02 is a potent cationic lipid for siRNA-Lipid Nanoparticles. SST-02 possesses a simple chemical structure and is synthesized just in one step. SST-02 showed an ID50 of 0.02 mg/kg in the factor VII (FVII) model. Rats administered with 3 mg/kg of SST-02

Elebsiran sodium is an siRNA targeting hepatitis B and D virus infections [1].

50-C2-C9-4tail is a chemical compound utilized in the creation of lipid nanoparticles (LNPs) for the efficient delivery of siRNA and mRNA, both in vitro and in vivo.

CL4H6, a pH-sensitive cationic lipid, serves as the primary constituent of lipid nanoparticles (LNPs). These LNPs are instrumental in targeting and delivering siRNA, leading to a potent gene-silencing response [1] [2].

SLN124 is a GalNac-siRNA that targets transmembrane serine protease 6 (Tmprss6) and consists of a trimeric GalNAc ligand bound to TMPRSS6-siRNA. SLN124 has the potential to be used as an siRNA agent to restore ferroregulatory expression and normalize iron homeostasis in β-depletion.

Dios-Arg is a steroid-based cationic lipid that contains a diosgenin skeleton coupled to an L-arginine head group. It forms a complex with plasmid DNA and decreases plasmid DNA migration in an agarose-gel retardant assay at charge ratio greater than or equal to 4. Dios-Arg facilitates transfection of plasmid DNA into H1299 and HeLa cells in the presence and absence of fetal bovine serum, an effect that is reversed by the lipid raft-mediated endocytosis inhibitor methyl-β-cyclodextrin and the caveolae-mediated endocytosis inhibitor genistein . It has been used, coupled to 1,2-dioleoyl-sn-glycero-3-PE , to bind siRNA and plasmid DNA to form cationic lipid nanoparticles for intracellular transport. Dios-Arg is cytotoxic to H1299 and HeLa cells (IC50s = 83.5 and 74.1 μg/ml, respectively).

98N12-5, a novel multi-tail ionizable lipid, demonstrates efficient in vivo siRNA delivery to the liver.

MVL5, a non-degradable multivalent cationic lipid, serves as a highly efficient vector for both DNA and siRNA [1].

ATU027, a small interfering RNA (siRNA), silences protein kinase N3 (PKN3) expression in the vascular endothelium. It has been demonstrated to thwart local tumor invasion, as well as lymph node and pulmonary metastasis, in mouse cancer models.

Bevasiranib, a siRNA, is specifically engineered to suppress the expression of genes responsible for the production of vascular endothelial growth factor (VEGF), a critical factor in the development of choroidal neo-vascularization (CNV), which precedes wet age-related macular degeneration (wet AMD).

Di-Pal-MTO, a lipid derivative synthesized from palm oil by integrating the anticancer agent mitoxantrone (MTO) with palmitoleic acid, exhibits enhanced anticancer activity and efficient siRNA cell delivery when nanoparticles of mono-Pal-MTO and di-Pal-MTO are combined at a molar ratio of 1:1 [1].

Cemdisiran, a conjugated siRNA with N-acetylgalactosamine (GalNAc), targets the liver to inhibit the production of complement 5 (C5) protein, serving as a potential therapeutic agent for the study of complement-mediated diseases.

DMHAPC-Chol is a cationic cholesterol. Liposomes containing DMHAPC-chol have been used for DNA plasmid delivery in vitro and in vivo in a B16-F10 mouse xenograft model. Liposomes containing DMHAPC-chol are cytotoxic to B16-F10 cells when used at lipid concentrations greater than 20 μM. DMHAPC-Chol, as part of a lipoplex with DOPE , has also been used to deliver DNA into mouse lung via intratracheal injection, resulting in a heterogeneous distribution in the bronchi and bronchioles, and to deliver VEGF siRNA into A431 and MDA-MB-231 cells, which secrete VEGF.

SSPalmO-Phe is a proton-activated, ionizable cationic lipid-like material featuring self-degradable, disulfide-cleavable (SS-cleavable) properties. It is utilized in conjunction with other lipids to create lipid nanoparticles (LNPs) for drug delivery purposes.

LNP Lipid-8 (11-A-M), an ionizable lipid, is utilized in lipid nanoparticles (LNP) for siRNA delivery to T cells without ligand targeting. When loaded with GFP siRNA (siGFP), it significantly silences the GFP gene in a mouse model [1].

Tivanisiran (SYL1001) is a siRNA developed to inhibit transient receptor potential vanilloid 1 (TRPV1) expression, aimed at investigating its role in dry eye disease research.

AZD-CO-Ph-PEG4-Ph-CO-AZD is a bis-β-lactam linker frequently used in the synthesis of antibody-siRNA conjugates.

Inclisiran (ALN-PCSsc) is a double-stranded small interfering RNA (siRNA) compound that effectively suppresses PCSK-9 transcription, showing promise in the investigation of hyperlipidemia and cardiovascular disease (CVD).

Givosiran (ALN-AS1) is a small interfering RNA (siRNA) compound that specifically targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA (mRNA) to downregulate its expression, thereby inhibiting the accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels, with significant potential for further research on acute intermittent porphyria.

HAPC-Chol is a cationic cholesterol. HAPC-Chol, as part of a lipoplex with DOPE , has been used for siRNA delivery and gene silencing in MCF-7 cells in a luciferase assay without affecting cell viability. It has also been used to deliver siRNA into mice via intravenous injection, resulting in HAPC-chol accumulation in the lungs.

DOTMA (N-(1-(2,3-dioleyloxy)propyl)-N,N,N-trimethylammonium) is a tetra-methylated DOTA analogue. DOTMA is a cationic lipid and can be used as a non-viral vector for gene therapy. It has been used as a component in liposomes that can be used to encapsulate siRNA, microRNAs, and oligonucleotides and for gene transfection in vitro. It exhibits effective in vitro and in vivo gene transfection. DOTMA induces a positive charge on the liposomes and thus promotes efficient liposome- cell membrane interaction.

Olpasiran, a small interfering RNA, reduces lipoprotein(a) synthesis in the liver.