Dystrophin Modulator

Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research.

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research.

TG693 is an orally active inhibitor of CLK1. TG693 regulates the mutated exon 31 of the dystrophin gene in vivo. TG693 is used in Duchenne muscular dystrophy (DMD) research.

Viltolarsen sodium scrambled negative control is the sequence scrambled negative control of Viltolarsen sodium.

AHR antagonist 8 (compound SG-02) is a regulator of utrophin, a homolog of dystrophin, and an AhR antagonist (Kd: 41.68 nM). Studies have shown that 800 nM of AHR antagonist 8 can upregulate utrophin by 2.7 times. AHR antagonist 8 also stimulates increased MyHC expression, suggesting that it has the potential to enhance myogenesis. After ADME evaluation, AHR antagonist 8 also has a certain oral bioavailability.

RTC14 is a read-through compound (RTC) that can induce ribosomes to bypass nonsense mutations in mRNA and allow the production of full-length functional proteins. RTC14 has the potential to be used in the research of various genetic disorders, such as nonsense mutations in the ataxia-telangiectasia mutated (ATM) gene and the dystrophin gene.