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Comment
. 2014 May 15;123(20):3068-9.
doi: 10.1182/blood-2014-04-565507.

A David promoter with Goliath strength

David M Markusic  1 , Roland W Herzog  1
Affiliations
Comment

A David promoter with Goliath strength

David M Markusic et al. Blood. .

Abstract

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.

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