To verify that the SAT1 disruption cassettes were successfully excised from GDH2/gdh2 and GDH3/gdh3 strains, genomic PCR was used with primers C1 and C4 to amplify the region between the upper end of 5 flank sequences to the lower end of 3 flank sequences to make sure that both alleles of each gene were removed (Supplementary Fig
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The Cranbury, New Jerseybased biotech announced FDA acceptance of its Biologics License Application for RP-L201 (marnetegragene autotemcel), an investigational lentiviral vector (LV)-based gene therapy for LAD-I, a rare, genetic immune disorder that predisposes patients to frequent and often fatal infections
IPR003115) but that lacked the strictly conserved SRX-N terminal binding motif F ( S / G ) GCHR required for catalytic activity (Supplementary file 1, Table S4)
