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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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Brown, Harrison C., Zakas, Philip M., George, Stephan N., Parker, Ernest T., Spencer, H. Trent, Doering, Christopher B.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5852392/
https://ncbi.nlm.nih.gov/pubmed/29552578
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.01.004
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