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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Detalles Bibliográficos
Autores principales: Brown, Harrison C., Zakas, Philip M., George, Stephan N., Parker, Ernest T., Spencer, H. Trent, Doering, Christopher B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5852392/
https://www.ncbi.nlm.nih.gov/pubmed/29552578
http://dx.doi.org/10.1016/j.omtm.2018.01.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5852392/
https://www.ncbi.nlm.nih.gov/pubmed/29552578
http://dx.doi.org/10.1016/j.omtm.2018.01.004

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