GJ-103 Sodium salt

GJ103 sodium salt is an active, water-soluble analog of the read-through compound GJ072. Chemical-induced read through of premature stop codons might be exploited as a potential treatment strategy for genetic disorders caused by nonsense mutations. It induces ATM kinase activity in A-T cells carrying different homozygous nonsense mutations; this analog is also active in A-T cells containing TAG or TAA stop codons.