… is similar to wild-type FIX but FVIIIa-dependent hyperactivity of R338L-Padua is the result of
a … AAV plasmid vectors contained a liver-specific promoter (α1-antitrypsin [AAT]) to express a …

We established a semi-high-throughput in vivo screening platform using hyperactive piggyBac
(hyPB) transposons (designated as PB-miR) to identify microRNAs (miRs) that inhibit …

… factor IX (FIX), … design of stronger synthetic liver-specific promoters, codon-optimized
F8 and F9 cDNAs, and use of engineered F8 (B-domain deleted FVIII variants) and F9 (hyperactive …

… trials for hemophilia B harnessed factor IX (FIX) expression from liver-specific promoters, ie
… computational methods, which explicitly consider evolutionarily conserved transcription factor …

… liver-specific or cardiac-specific CRMs 41,49,50 . However, in the present study we further
refined this computational … biochemical features associated with transcription regulation and/or …

… is an investigational factor IX (FIX) gene transfer product that utilizes the adeno-associated
virus (AAV) 5 vector with a liver-specific promoter and a hyperactive FIX transgene. Here, the …

… This product is based on an AAV5 vector expressing a hyper-active factor IX (FIX)
transgene … are designed to encapsulate an FVIII or FIX transgene downstream of a liver-specific …

… blood clotting disorder due to the deficiency of factor IX (FIX) induced by mutations in the …
expressing mutated cFIX under the control of the liver-specific human α1-antitrypsin (hAAT) …

… data demonstrating stable factor VIII (FVIII) and factor IX (FIX) … the hybrid liver-specific
promoter (HLP) , was created for use … Further, AAV2/8 vectors expressing FVIII and a hyperactive …

… two liver-specific promoters in this study and designed two … P3 promoter was proved to be
robust and liver-specific. It has … promoter is a particularly important factor in our repair strategy. …