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- W3082495512 abstract "Adeno-associated virus (AAV)-based gene therapy is currently limited by (1) decline in therapeutic gene expression over time, (2) immune cell activation and (3) neutralization by pre-existing antibodies. Hence, studying the interaction of AAV vectors with various cellular pathways during the production and transduction process is necessary to overcome such barriers. Post-translational modifications (PTM) of AAV vectors during the production and transduction process is known to limit its transduction efficiency and further evoke the immune response. Further, AAV vectors are known to trigger cellular stress, resulting in an upregulation of distinct arms of the unfolded protein response (UPR) pathway. Recognition of the AAV genome by Toll-like receptor-9 triggers the myeloid differentiation primary response signaling cascade for innate (IL-6, IFN-α, IFN-β) and adaptive (CD8+ T-cell, B-cell) immune response against the viral capsid and the transgene product. Herein, we highlight a potential intersection of the UPR, PTMs, and intracellular trafficking pathways, which could be fine-tuned to augment the outcome of AAV-based gene delivery." @default.
- W3082495512 created "2020-09-08" @default.
- W3082495512 creator A5046861109 @default.
- W3082495512 creator A5085893176 @default.
- W3082495512 date "2020-08-28" @default.
- W3082495512 modified "2023-09-25" @default.
- W3082495512 title "Gene Therapy: Contest between Adeno-Associated Virus and Host Cells and the Impact of UFMylation" @default.
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- W3082495512 doi "https://doi.org/10.1021/acs.molpharmaceut.0c00512" @default.
- W3082495512 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/32857512" @default.
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