These results show that codon optimization, a lentiviral delivery system, and clonal selection together enable the establishment of stable human cell lines capable of producing high levels of biologically active, synthetic recombinant GCase in vitro.
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codon optimization
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This review considers existing modern approaches aimed to overcome both pre-existing natural immunity and immunity obtained after the administration of a gene therapy drug, which include various modifications of the viral drug (capsid modification, codon optimization)...
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Codon optimization, lentiviral delivery, and clonal selection together enabled establishment of stable human cell lines producing high levels of biologically active synthetic recombinant GCase in vitro.
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These results show that codon optimization, a lentiviral delivery system, and clonal selection together enable the establishment of stable human cell lines capable of producing high levels of biologically active, synthetic recombinant GCase in vitro.
The review identifies capsid modification, codon optimization, empty capsid traps, corticosteroids, immune response inhibitors, nanoparticles, and IgG-degrading enzymes as approaches to overcome pre-existing and post-administration AAV immunity.