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Fig. 3 | Cell & Bioscience

Fig. 3

From: Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapy

Fig. 3

Schematic illustration of permanent correction of CFTR mutations in CF airway epithelia. (Left panel) HD-Ad vector particles carrying the Cas9/sgRNA genes and donor DNA with homology arms are delivered into airway epithelial cells, including airway basal stem cells. (Middle panel) Following the vector genome reach the nuclei of target cells, the Cas9 and its corresponding sgRNA are expressed and they work together to generate a double-stranded DNA break at the target site where the donor DNA integrates through homologous DNA strand exchanges at both left and right homology arms. If the donor DNA integration does not happen in a cell, the break is repaired through non-homologous end joining. (Right panel) The donor DNA integration compromises the vector genome integrity, leading the degradation of the rest of the vector genome and thus eliminating the undesirable expression of the Cas9 protein. While the terminally differentiated epithelial cells are eventually replaced, the permanently gene-corrected basal stem cells will be differentiated into other epithelial cells, thus perpetuating the therapeutic effects of gene correction

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