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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
10 Results for "
targeted gene repair
" in MedChemExpress (MCE) Product Catalog:
Products (10)
10 Results for "targeted gene repair" 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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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) .
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TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle .
Cat. No.: HY-P5244
CAS No.: 1185583-20-3
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Cat. No.: HY-174511
Human TP53 mRNA encodes the human tumor protein p53 (TP53) protein, a tumor suppressor protein containing transcriptional activation, DNA binding, and oligomerization domains. TP53 responds to diverse cellular stresses to regulate expression of target genes, thereby inducing cell cycle arrest, apoptosis, senescence, DNA repair, or changes in metabolism.
Cat. No.: HY-P990873
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Cat. No.: HY-L244
761 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 761 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-150279R
CAS No.: 2565638-16-4
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PolQi2 (Standard) is the analytical standard of PolQi2 (HY-150279). This product is intended for research and analytical applications. 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 .
Cat. No.: HY-180944
CAS No.: 667889-47-6
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BE2012, SR8278 (HY-14415) derivative, is a potent and selective REV-ERBα/β Antagonist with EC50 values of 0.285 and 0.346 μM. BE2012 binds to the ligand-binding domain (LBD) of REV-ERB, preventing the receptor from recruiting co-inhibitory factors and thereby releasing the transcriptional repression on downstream target genes. BE2012 can upregulate the expression of myogenic transcription factors Myf5 and Myod, promoting muscle regeneration and repair in acute muscle injury micemodels .
Cat. No.: HY-L024
931 compounds
A histone modification, a covalent post-translational modification (PTM) to histone proteins, includes methylation, phosphorylation, acetylation, ubiquitylation, and sumoylation, etc. In general, histone modifications are catalyzed by specific enzymes that act predominantly at the histone N-terminal tails involving amino acids such as lysine or arginine, as well as serine, threonine, tyrosine, etc. The PTMs made to histones can impact gene expression by altering chromatin structure or recruiting histone modifiers. Histone modifications act in diverse biological processes such as transcriptional activation/inactivation, chromosome packaging, and DNA damage/repair. Deregulation of histone modification contributes to many diseases, including cancer and autoimmune diseases.
MCE owns a unique collection of 931 bioactive compounds targeting Epigenetic Reader Domain, HDAC, Histone Acetyltransferase, Histone Demethylase, Histone Methyltransferase, Sirtuin, etc. Histone Modification Research Compound Library is a useful tool for histone modification research and drug screening.
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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
