“Combining the newer base editors with dual splice editing skipped the exon at a much better rate than we were previously able to with any of the available methods. We were able to show that not ...

This step occurs by transesterification; in this case, an OH group at the 3′ end of the exon attacks the phosphodiester bond at the 3′ splice site. The adjoining exons are covalently bound ...

GenAssist’s IND application for GEN6050X receives US FDA clearance, a globally first-in-class base editing drug for duchenne muscular dystrophy: Suzhou, China Friday, March 7, 2 ...

Intronic primers flanking exon 9 were designed using the PAC sequence available in the Genbank sequence database (clone DJ0054D12; accession number AC006342). PCR amplification of an approximate 6 ...