Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A

Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A

Potency is a key optimization parameter for hemophilia A gene therapy product candidates. Optimization strategies include promoter engineering to increase transcription, codon optimization of mRNA to improve translation, and amino-acid substitution to promote secretion. Herein, we describe both rati...

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Bibliographic Details
Main Authors: Harrison C. Brown, Philip M. Zakas, Stephan N. George, Ernest T. Parker, H. Trent Spencer, Christopher B. Doering
Format: Article
Language:English
Published: Elsevier 2018-06-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118300056

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http://www.sciencedirect.com/science/article/pii/S2329050118300056

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