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However, many factors, including their low stability, short half-life, high immunogenicity, tissue targeting, cellular uptake, and endosomal escape, hamper the delivery and clinical application of NADs

Rajagopalan, S., Andreeva, A., Teufel, D

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Plasmid for single-stranded adeno-associated viral vectors 2/8 The plasmid pAAV-MCS-hAAT/EGFP was designed for maximum transgene expression and includes the hepatic locus control region from the ApoE gene (ApoE-HCR), a liver-specific 1-antitrypsin promoter (hAAT promoter), coagulation factor IX intron A (FIX intron A), a 3' mRNA transcription termination/polyadenylation signal (bghpA), and a reporter gene encoding enhanced green fluorescent protein (EGFP), as previously described (Miao et al