File:MUS-64-413-g002.jpg
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English: After intravenous administration of onasemnogene abeparvovec, the SMN transgene is maintained as a non‐integrating episome in the nuclei of target cells. A, Attachment and entry. Cell‐surface attachment of rAAV is mediated by primary glycan receptors and stabilized by proteinaceous secondary receptors. B, Internalization. AAV‐based vectors utilize multiple endocytic routes during their journey towards the nucleus. C, Sorting steps. The rAAV capsid undergoes substantial conformational changes in the sorting compartments (endosomes and the Golgi). D, Cytoplasmic escape and nuclear import. After escaping the sorting compartments, vector particles accumulate around the perinuclear space and enter the nucleus via nuclear pore complexes. E, Genome release and transgene expression. Inside the nucleus, the vector genome is released and transcribed, resulting in the expression of the transgene. Abbreviations: dsDNA, double‐stranded DNA; GEEC, glycosylphosphatidylinositol‐anchored protein (GPI‐AP)‐enriched compartment; scAAV, self‐complementary AAV; ssAAV, single‐stranded AAV; TGN, trans‐Golgi network. Reprinted from Trends in Molecular Medicine, Dhungel BP, Bailey CG, and Rasko JEJ, Journey to the center of the cell: tracing the path of AAV transduction, p4, 2020, with permission from Elsevier |
Date | |
Source | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518380/ |
Author | Elizabeth A. Kichula, Crystal M. Proud,Michelle A. Farrar, Jennifer M. Kwon, Kayoko Saito, Isabelle Desguerre, and Hugh J. McMillan |
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English: This file is licensed CC BY-NC-ND 4.0
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current | 19:48, 21 January 2023 | 709 × 935 (160 KB) | Ozzie10aaaa (talk | contribs) | Uploaded a work by Elizabeth A. Kichula, Crystal M. Proud,Michelle A. Farrar, Jennifer M. Kwon, Kayoko Saito, Isabelle Desguerre, and Hugh J. McMillan from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518380/ with UploadWizard |
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