A David promoter with Goliath strength.
In this issue of Blood, Nair et al report on a computationally derived hepatocyte-specific cis-regulatory module (HS-CRM8) to accomplish high tissue-specific expression in gene therapy for hemophilia. When HS-CRM8 is introduced upstream of a minimal liver-specific promoter in an adenoassociated virus (AAV) that expresses a codon-optimized hyperactive human factor IX (FIX) mutant (FIX Padua), it provides a >1 log increase in systemic FIX protein levels and supraphysiological activity over a range of vector doses.
UNIVERSITY OF FLORIDA.
David M
DM
Markusic
UNIVERSITY OF FLORIDA.
Roland W
RW
Herzog
D000818:Animals; D005164:Factor IX / Q000235:genetics*; D005822:Genetic Vectors / Q000627:therapeutic use*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans; D008099:Liver / Q000378:metabolism*; D050436:Regulatory Elements, Transcriptional
D005164:Factor IX