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Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
27 Results for "
gene edited
" in MedChemExpress (MCE) Product Catalog:
Products (27)
27 Results for "gene edited" in MCE Product Catalog:
3
3 Cited Publications
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PolQi2 is a PolΘ inhibitor that targets and inhibits alt-EJ (alternative end-joining) repair by inhibiting the helicase domain at the N-terminus of PolΘ. PolQi2 enhances the precision and integration efficiency of gene editing at different loci and in various cell lines. Furthermore, the combined use of PolQi2 with DNA-PK inhibitors reduces the off-target effects of Cas9. PolQi2 can be used in gene editing research .
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1 Cited Publications
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UNC9512 is a selective 53BP1 inhibitor with an IC50 of 0.46 μM, and a Kd values of 0.17 μM. UNC9512 binds 53BP1 and its tandem Tudor domain, disrupts histone H4 interaction, and inhibits 53BP1 activity. UNC9512 can be used as a probe for DNA damage repair and Gene editing .
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PolQi1 is a selective inhibitor targeting the Polθ domain of DNA polymerase. PolQi1 inhibits the Polθ-mediated microhomology end joining (TMEJ/alt-EJ) pathway, reducing insertion/deletion (Indels) and imprecise editing events during DNA repair. PolQi1 can enhance the efficiency and accuracy of homology-directed repair (HDR) or Prime editing, and reduce off-target effects; and in combination with DNA-PK inhibitor AZD-7648 (HY-111783), exert efficient genome editing capabilities with dual pathway regulation. PolQi1 can be mainly used in gene editing research (such as CRISPR-Cas9 or Prime editing system optimization) to improve the precision editing efficiency of difficult-to-edit cells (such as primary hepatocytes and mouse embryos) .
Cat. No.: HY-W010744
CAS No.: 93778-57-5
Synonyms: 5'-O-DMT-dI; 2'-Deoxy-5'-O-DMT-inosine
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DMT-dI (5'-O-DMT-dI) is a deoxyribonucleoside containing a hypoxanthine base. DMT-dI can be used to prepare convertible nucleoside derivatives to prepare modified oligonucleotides complementary to target genes for gene editing. DMT-dI can be used to study various conditions, disorders or diseases modified by adenosine .
Cat. No.: HY-147412
CAS No.: 2642382-41-8
Synonyms: QR-421a
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Ultevursen (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .
Cat. No.: HY-172526
CAS No.: 3082361-42-7
4A3-SCC-10 is a biodegradable ionizable lipid containing disulfide bonds. 4A3-SCC-10 can be used to prepare lipid nanoparticles (LNPs) for the delivery of mRNA in vitro and in vivo. 4A3-SCC-10 can be used for broad applications such as gene editing, vaccine, and cancer detection .
Cat. No.: HY-E70220
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AsCas12a Nuclease is a nuclease, and can specifically cutting double-stranded DNA. AsCas12a Nuclease can be used for gene edited study .
Cat. No.: HY-159709
CAS No.: 2765519-47-7
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VL-422 is an ionizable cationic lipid. VL-422 delivers CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA to enable in vitro and in vivo gene editing. LNPs containing VL-422 loaded with Cas9 mRNA and sgRNA targeting the ANGPTL3 gene induce the deletion of premature stop codons within the ANGPTL3 gene in the liver of cynomolgus monkeys. Loss-of-function of ANGPTL3 leads to decreased levels of LDL, HDL and cholesterol in plasma. The VL-422 delivery system can be used for the research of gene editing strategies targeting lipid metabolism diseases .
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RNA, (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5C-m5U-m5U-m5C-G-G-A-G-A-A-A-m5U-m5U-m5U-A-A-A-m5U-m5C), sodium salt
Cat. No.: HY-147412A
Synonyms: QR-421a sodium
Cat. No.: HY-176754
CAS No.: 1872269-57-2
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TCL065 is an ionizable lipid with a pKa of 6.3. TCL065 can be used to generate lipid nanoparticles (LNPs) for the delivery of mRNA as well as single-guide RNA (sgRNA) both in vitro and in vivo. TCL065-containing LNPs can studied in research for Duchenne muscular dystrophy and gene-editing technologies .
Cat. No.: HY-171953
CAS No.: 3097638-13-3
THP1 Lipid is an ionizable lipidoid. THP1 Lipid can be used to synthesize lipid nanoparticles (LNPs) for delivering mRNA to muscles with minimal toxicity and editing genes in specific liver tissues in tdTomato transgenic mice model. THP1 Lipid can be used for vaccine delivery and CRISPR/Cas9-mediated gene editing research .
Cat. No.: HY-165604
CAS No.: 2639634-82-3
C14-490, an ionizable cationic lipid (pKa = 5.94), is used for the synthesis of lipid nanoparticles (LNPs). C14-490 LNPs is used as the basis for subsequent study of in utero gene editing in HSCs. C14-490 LNPs are used to encapsulate SpCas9 mRNA and TTR sgRNA using optimized B5 formulation parameters and surface conjugation to CD45 antibody F(ab’)2 fragments-Systematically optimized Targeted Editing Machinery LNPs (STEM LNPs) .
Cat. No.: HY-L244
756 compounds
In this era of rapid advancement in gene-editing technology, the CRISPR-Cas system, with its powerful programmability, is leading a transformation in life sciences research. It enables efficient and precise targeted modification of an organism's genome, providing a robust tool for studying gene function, treating genetic diseases, and improving crop varieties. However, bottlenecks such as insufficient editing efficiency, low homologous directed repair efficiency, and potential off-target risks remain major challenges in achieving precise genetic modifications and developing gene therapies.
To overcome these limitations, the MCE High-Efficiency Gene Editing Compound Library systematically includes 756 small molecules that are known or have the potential to enhance gene-editing efficiency. These compounds work by targeting and modulating the DNA damage repair network, mechanistically inhibiting non-homologous end joining, promoting homologous directed repair, or regulating chromatin states and cellular responses, thereby significantly optimizing editing outcomes. This library is suitable for developing "CRISPR-small molecule" combination therapy strategies, improving gene-editing efficiency, and providing a powerful tool for in-depth research into the mechanisms of DNA damage repair in gene editing.
Cat. No.: HY-P11639
CAS No.: 380480-77-3
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Cat. No.: HY-185751
CAS No.: 2760467-77-2
10A1P16 is an ionizable phospholipid composed of a tertiary amine, a phosphate group and three hydrophobic tails. As a spleen-selective RNA delivery vector, 10A1P16 successfully transfects approximately 30% of splenic macrophages and 6% of splenic B cells in vivo. 10A1P16 can be used to prepare spleen-selective 10A1P16-MDOA lipid nanoparticles, which effectively mediate mRNA delivery and gene editing in mouse models. This delivery system retains potent spleen-targeted mRNA delivery efficacy and can be applied to ovarian cancer research .
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Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
