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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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Publicado no: | Mol Ther Methods Clin Dev |
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Main Authors: | , , , , , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
American Society of Gene & Cell Therapy
2018
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Assuntos: | |
Acesso em linha: | 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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