editing

8-Azaadenosine, a powerful ADAR1 (ADAR1) and A-to-I editing inhibitor, effectively blocks RNA editing and suppresses proliferation, 3D growth, invasion, and migration in thyroid cancer cells.

BRD20322 is a novel potent inhibitor of spCas9 that disrupts spCas9-DNA binding and exerts dose and temporal control of spCas9 in human cell lines. It reduces off-target DNA editing events, enhancing the precision and safety of CRISPR-Cas9 gene editing.

(iso)-BRD20322 is an isomer of BRD20322, a novel potent inhibitor of spCas9 that disrupts the binding of spCas9 to DNA and reduces non-specific DNA editing events, thereby improving the safety and accuracy of the CRISPR-Cas9 gene editing system.

93-O17O is a chalcogen-containing cationic lipidoid used in the generation of lipid nanoparticles (LPNs). LPNs containing 93-O17O localize to the spleen after intravenous injection into mice and have been utilized for delivering Cre recombinase, ribonucleoproteins for genome editing, and intratumoral delivery of cGAMP to enhance cross-presentation of tumor antigens. [1,2,3]

93-O17S is a chalcogen-containing cationic lipidoid. It has been used in the synthesis of lipid nanoparticles (LPNs) for the delivery of Cre recombinase and ribonucleoproteins for genome editing in mice. LPNs containing 93-O17S have been utilized for the intratumoral delivery of cGAMP to enhance cross-presentation of tumor antigens and stimulation of interferon genes [STING] activation in a B16 F10 murine melanoma model.

SF2523 is a highly selective and potent inhibitor (SF2523) with improved physicochemical properties, excellent aqueous solubility, and biological efficacy. It is typically used in genome editing [CRISPR/Cas Solution] technology and can effectively enhance the efficiency of this process.

BAMEAO16B is a lipid nanoparticle that incorporates disulfide bonds for efficient delivery of Cas9 mRNA and sgRNA into cells. In the intracellular environment, BAMEAO16B responds to reduction by releasing RNA, facilitating genome editing. This compound is valuable for gene editing research purposes.

Cas9-IN-3 is a potent inhibitor of Cas9 with an IC50 of 28 μM. The CRISPR Cas systems have revolutionized gene editing in several species [1].

8-Azanebularine, a chemical compound substituting hydrogen for the C6 amino group, effectively inhibits the ADAR2 reaction at elevated concentrations (IC50=15 mM), and exhibits high-affinity binding (KD=2 nM) when incorporated into an RNA structure recognized by human ADAR2. This characteristic makes 8-Azanebularine valuable for researching the ADAR-catalyzed RNA-editing reaction.

ZA3-Ep10, a zwitterionic lipid, is utilized in the formulation of lipid nanoparticles for RNA delivery in vivo and non-viral CRISPR Cas gene editing applications.

306-N16B is a lipid nanoparticle designed for the systemic co-delivery of Cas9 mRNA and sgRNA, facilitating the transport of mRNA to pulmonary endothelial cells. This compound is advantageous for research focused on genome editing-based therapies [1].

UNC9512 is a potent antagonist of the methyl-lysine reader protein 53BP1, which can be utilized to investigate the function of 53BP1 in DNA repair, gene editing, and tumorigenesis [1].

Peptide A5K acetate (INF7-A5K-TAT acetate) is an amphiphilic peptide derived from the HA2-TAT fusion scaffold, designed to facilitate macromolecular transport in genome editing. Peptide A5K acetate non-covalently binds to CRISPR RNP (ribonucleoprotein complex) and delivers it into cells such as primary human T cells, B cells, and NK cells, enabling low-toxicity, multi-target, highly efficient, and precise genome editing.

9A1P9, a multi-tail ionizable cationic phospholipid, facilitates membrane destabilization and is applicable for CRISPR-Cas9 gene editing in mice [1].

NT1-O14B is a novel class of neurotransmitter tryptophan lipids (NT-lipidoids).NT1-O14B can be combined with small molecule drugs (e.g., AmB) and biomolecules (e.g., Tau-ASO) and gene editing proteins (GFP-Cre) to form new neurotransmitter-derived lipid nanoparticles (NT-LNPs) that have blood-brain-barrier (BBB) permeability. Enhanced lipid hydrophobicity and membrane fusion capabilities are commonly used for delivery of nucleic acids such as mRNA, siRNA and DNA.

PolQi2 is a Polθ inhibitor that suppresses the helicase activity of Polθ. It can be combined with AZD7648 to enhance HDR-mediated integration and editing accuracy.

FTT5 LLNs are functionalized lipid-like nanoparticles designed for in vivo mRNA delivery and base editing.

Si5-N14 is a key component of siloxane-linked lipid nanoparticles (SiLNP) with properties that enhance vascular repair and exhibit antitumor activity. In transgenic GFP mouse models, Si5-N14 mediates CRISPR-Cas9 editing. In Lewis lung carcinoma (LLC) tumor mouse models, it leads to the knockdown of vascular endothelial growth factor receptor 2 (VEGFR2), producing antitumor effects. Additionally, in mice with virus-induced lung injury, Si5-N14 facilitates the delivery of fibroblast growth factor-2 (FGF-2) mRNA, promoting vascular repair, oxygenation, and improved lung function. Si5-N14 shows potential for research in tumors, pneumonia, and cardiovascular diseases.

TCL065, an ionizable cationic lipid (pKa = 6.3), is utilized in the creation of lipid nanoparticles (LNPs) for the delivery of mRNA and single-guide RNA (sgRNA) both in vitro and in vivo. When LNPs with TCL065 encapsulate sgRNA targeting the donor site of dystrophin exon 45, along with those targeting the splicing acceptor site of dystrophin exon 45, dystrophin expression is restored in myoblasts derived from induced-pluripotent stem (iPS) cells from Duchenne muscular dystrophy (DMD) patients. Additionally, TCL065-containing LNPs encapsulating mRNA encoding Cas9 nuclease, combined with those containing sgRNA targeting the Rosa26 locus, have been applied for CRISPR-mediated gene editing in mouse gastrocnemius muscle.

AsCas12a Nuclease is a CRISPR nuclease capable of specifically cleaving double-stranded DNA. It is used in gene editing research.

C14-490 is an ionizable cationic lipid (pKa= 5.94) utilized in the synthesis of lipid nanoparticles (LNPs). These LNPs serve as a platform for subsequent gene editing studies in hematopoietic stem cells (HSCs) in utero. C14-490 LNPs encapsulate SpCas9 mRNA and TTR sgRNA, employing an optimized B5 formulation parameter, and are further enhanced by the surface attachment of CD45 antibody F(ab’)2 fragments to create Systemically Targeted Editing Mechanism LNPs (STEM LNPs).