gene edited

By Targets:	Bacterial (1) Biochemical Assay Reagents (2) DNA/RNA Synthesis (4) Fluorescent Dye (2) Liposome (8) Nucleoside Antimetabolite/Analog (2) Phosphoramidites (1)
By Research Areas:	Cancer (5) Inflammation/Immunology (2) Metabolic Disease (2) Neurological Disease (4)
Oligonucleotides:	Hypoxanthine (1) Cationic Lipids (6) Antisense Oligonucleotides (4) Phosphoramidites (1)

Targets Recommended: LAG-3 CGRP Receptor MALT1 Dan family RIG-I-like receptor (RLR) NEKs

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-150279

PolQi2 3 Publications Verification	DNA/RNA Synthesis	Others
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 .

HY-159078

PolQi1	DNA/RNA Synthesis	Cancer
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) .

HY-145795

OF-02	Liposome	Others
OF-02 is an ionizable lipid for mRNA delivery and a key component of lipid nanoparticles (LNPs). OF-02 is protonated in the acidic endosomal environment to promote endosomal escape of mRNA and efficiently induce protein expression in target cells. OF-02 relies on the pH-responsive membrane fusion properties to form a complex with mRNA and destroy the endosomal membrane structure to achieve mRNA release in the cytoplasm. OF-02 is mainly used in the development of mRNA vaccines, gene editing, and protein replacement therapy[1][2][3].

HY-150229

306-N16B	Liposome	Cancer
306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

HY-139306

BAMEA-O16B	Liposome	Inflammation/Immunology
BAMEAO16B is a lipid nanoparticle. BAMEAO16B integrated with disulfide bonds, can efficiently deliver Cas9 mRNA and sgRNA into cells while releasing RNA in response to the reductive intracellular environment for genome editing. BAMEAO16B can be used for the research of gene editing .

HY-156257

UNC9512 1 Publications Verification	DNA/RNA Synthesis	Cancer
UNC9512 is a selective 53BP1 inhibitor with an IC₅₀ of 0.46 μM, and a K_d 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 .

HY-W010744

DMT-dI 5'-O-DMT-dI; 2'-Deoxy-5'-O-DMT-inosine	Phosphoramidites Nucleoside Antimetabolite/Analog	Cancer
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 .

HY-147412

Ultevursen QR-421a	Nucleoside Antimetabolite/Analog DNA/RNA Synthesis	Neurological Disease
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 .

HY-172526

4A3-SCC-10	Liposome	Cancer
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 .

HY-E70220

AsCas12a Nuclease	Biochemical Assay Reagents	Others
AsCas12a Nuclease is a nuclease, and can specifically cutting double-stranded DNA. AsCas12a Nuclease can be used for gene edited study .

HY-159709

VL-422	Liposome	Metabolic Disease
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 .

HY-176754

TCL065	Liposome	Metabolic Disease Inflammation/Immunology
TCL065 is an ionizable lipid with a pK_a 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 .

HY-171953

THP1 Lipid	Liposome	Others
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 .

HY-145692

Cas9-IN-3	Bacterial	Others
Cas9-IN-3 is a potent Cas9 inhibitor (IC₅₀=28 μM) .

HY-165604

C14-490	Liposome	Others
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) .

HY-P11639

ppTG21	Biochemical Assay Reagents	Others
ppTG21 is an endosomolytic agent and inducer. ppTG21 can facilitate endosomal escape of Cas9-2lig-1NLS and Cas9-ASGPrL ribonucleoproteins (RNPs) to enable receptor-facilitated, cell-type specific gene editing in cancer cells without electroporation or transfection reagents. ppTG21 can be used for the research of gene editing .

HY-185504

Nexiguran	Others	Others
Nexiguran is one component of Nexiguran ziclumeran and also an sgRNA targeting the human TTR gene. Nexiguran ziclumeran (NTLA-2001) is a CRISPR gene-editing therapy that targets hepatic TTR, and it can also be used for research on transthyretin amyloidosis .

HY-147412D

FAM labled Ultevursen sodium	Fluorescent Dye	Neurological Disease
FAM labled Ultevursen sodiumis a FAM labled Ultevursen sodium (HY-147412A). Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

HY-147412E

Cy3 labled Ultevursen sodium	Fluorescent Dye	Neurological Disease
Cy3 labled Ultevursen sodium is a Cy3 labled Ultevursen sodium (HY-147412A). Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

Cat. No.	Product Name

HY-L244

High-Efficiency Gene Editing Compound Library	730 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 730 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.

High-Efficiency Gene Editing Compound Library

730 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 730 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.

HY-L251

Ionizable Lipid Compound Library	95 compounds
Ionizable lipids are a class of specialized, functional lipid molecules with pH-sensitive charge characteristics. They are primarily divided into two major categories: ionizable cationic lipids and ionizable anionic lipids, though the term typically specifies ionizable cationic lipids within the biomedical field. Structurally, these lipids consist of an ionizable hydrophilic headgroup, a biodegradable linker, and hydrophobic tails. Their primary application is serving as the key delivery vehicle in lipid nanoparticles (LNPs) to encapsulate negatively charged nucleic acid macromolecules, such as mRNA vaccines, siRNA therapeutics, and CRISPR gene-editing components. In a physiological, neutral environment, they remain electrically neutral to minimize systemic toxicity and prolong circulation time. Upon entering the acidic microenvironment of cellular endosomes, however, they undergo protonation to become positively charged, thereby inducing membrane fusion and enabling the highly efficient intracellular release of the nucleic acid cargo. Consequently, they serve as the technological cornerstone for bringing nucleic acid therapies into clinical application. To accelerate the translational process of cutting-edge nucleic acid drugs, MCE has meticulously constructed an ionizable lipid compound library containing 95 high-performance molecules, aiming to provide researchers and pharmaceutical professionals with a high-throughput, multi-dimensional lipid screening platform.

Ionizable Lipid Compound Library

95 compounds

Ionizable lipids are a class of specialized, functional lipid molecules with pH-sensitive charge characteristics. They are primarily divided into two major categories: ionizable cationic lipids and ionizable anionic lipids, though the term typically specifies ionizable cationic lipids within the biomedical field. Structurally, these lipids consist of an ionizable hydrophilic headgroup, a biodegradable linker, and hydrophobic tails. Their primary application is serving as the key delivery vehicle in lipid nanoparticles (LNPs) to encapsulate negatively charged nucleic acid macromolecules, such as mRNA vaccines, siRNA therapeutics, and CRISPR gene-editing components. In a physiological, neutral environment, they remain electrically neutral to minimize systemic toxicity and prolong circulation time. Upon entering the acidic microenvironment of cellular endosomes, however, they undergo protonation to become positively charged, thereby inducing membrane fusion and enabling the highly efficient intracellular release of the nucleic acid cargo. Consequently, they serve as the technological cornerstone for bringing nucleic acid therapies into clinical application.

To accelerate the translational process of cutting-edge nucleic acid drugs, MCE has meticulously constructed an ionizable lipid compound library containing 95 high-performance molecules, aiming to provide researchers and pharmaceutical professionals with a high-throughput, multi-dimensional lipid screening platform.

Cat. No.	Product Name	Type

HY-145795

OF-02	Biochemical Assay Reagents
OF-02 is an ionizable lipid for mRNA delivery and a key component of lipid nanoparticles (LNPs). OF-02 is protonated in the acidic endosomal environment to promote endosomal escape of mRNA and efficiently induce protein expression in target cells. OF-02 relies on the pH-responsive membrane fusion properties to form a complex with mRNA and destroy the endosomal membrane structure to achieve mRNA release in the cytoplasm. OF-02 is mainly used in the development of mRNA vaccines, gene editing, and protein replacement therapy[1][2][3].

OF-02

Biochemical Assay Reagents

OF-02 is an ionizable lipid for mRNA delivery and a key component of lipid nanoparticles (LNPs). OF-02 is protonated in the acidic endosomal environment to promote endosomal escape of mRNA and efficiently induce protein expression in target cells. OF-02 relies on the pH-responsive membrane fusion properties to form a complex with mRNA and destroy the endosomal membrane structure to achieve mRNA release in the cytoplasm. OF-02 is mainly used in the development of mRNA vaccines, gene editing, and protein replacement therapy[1][2][3].

HY-150229

306-N16B	Biochemical Assay Reagents
306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

306-N16B

Biochemical Assay Reagents

306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

HY-139306

BAMEA-O16B	Biochemical Assay Reagents
BAMEAO16B is a lipid nanoparticle. BAMEAO16B integrated with disulfide bonds, can efficiently deliver Cas9 mRNA and sgRNA into cells while releasing RNA in response to the reductive intracellular environment for genome editing. BAMEAO16B can be used for the research of gene editing .

Cat. No.	Product Name	Target	Research Area

HY-P11639

ppTG21	Biochemical Assay Reagents	Others
ppTG21 is an endosomolytic agent and inducer. ppTG21 can facilitate endosomal escape of Cas9-2lig-1NLS and Cas9-ASGPrL ribonucleoproteins (RNPs) to enable receptor-facilitated, cell-type specific gene editing in cancer cells without electroporation or transfection reagents. ppTG21 can be used for the research of gene editing .

Cat. No.	Product Name

HY-KE7066

FnCas12a
FnCas12a(Cpf1), is an RNA-guided, DNA-editable recombinant endonuclease that can be used for gene editing and detection.

HY-K2028

OptiLNP Gene Editing Kit (Immune Cells)
MCE OptiLNP Gene Editing Kit (Immune Cells) is a ready-to-use transfection reagent based on LNP technology. It is designed for the efficient co-transfection of Cas9 mRNA and sgRNA during gene editing inimmune cells.

HY-K2027

OptiLNP Gene Editing Kit
MCE OptiLNP Gene Editing Kit is a ready-to-use transfection reagent based on LNP technology. It is designed for the efficient co-transfection of Cas9 mRNA and sgRNA during gene editing in common cells (adherent or suspension cells).

Cat. No.	Product Name		Classification

HY-145795

OF-02		Cationic Lipids
OF-02 is an ionizable lipid for mRNA delivery and a key component of lipid nanoparticles (LNPs). OF-02 is protonated in the acidic endosomal environment to promote endosomal escape of mRNA and efficiently induce protein expression in target cells. OF-02 relies on the pH-responsive membrane fusion properties to form a complex with mRNA and destroy the endosomal membrane structure to achieve mRNA release in the cytoplasm. OF-02 is mainly used in the development of mRNA vaccines, gene editing, and protein replacement therapy[1][2][3].

HY-150229

306-N16B		Cationic Lipids
306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

HY-139306

BAMEA-O16B		Cationic Lipids
BAMEAO16B is a lipid nanoparticle. BAMEAO16B integrated with disulfide bonds, can efficiently deliver Cas9 mRNA and sgRNA into cells while releasing RNA in response to the reductive intracellular environment for genome editing. BAMEAO16B can be used for the research of gene editing .

HY-W010744

DMT-dI 5'-O-DMT-dI; 2'-Deoxy-5'-O-DMT-inosine		Phosphoramidites Hypoxanthine
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 .

HY-147412

Ultevursen QR-421a		Antisense Oligonucleotides
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 .

HY-159709

VL-422		Cationic Lipids
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 .

HY-147412A

Ultevursen sodium QR-421a sodium		Antisense Oligonucleotides
Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

HY-176754

TCL065		Cationic Lipids
TCL065 is an ionizable lipid with a pK_a 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 .

HY-171953

THP1 Lipid		Cationic Lipids
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 .

HY-147412D

FAM labled Ultevursen sodium		Antisense Oligonucleotides
FAM labled Ultevursen sodiumis a FAM labled Ultevursen sodium (HY-147412A). Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

HY-147412E

Cy3 labled Ultevursen sodium		Antisense Oligonucleotides
Cy3 labled Ultevursen sodium is a Cy3 labled Ultevursen sodium (HY-147412A). Ultevursen sodium (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 sodium 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 sodium 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 sodium can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

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