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Review
. 1999 Feb;103(4):441-5.
doi: 10.1172/JCI6330.

Status of gene therapy for cystic fibrosis lung disease

Affiliations
Review

Status of gene therapy for cystic fibrosis lung disease

R C Boucher. J Clin Invest. 1999 Feb.
No abstract available

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Figures

Figure 1
Figure 1
Barriers to vector-mediated gene transfer in WD columnar airway epithelial cells. The failure of vectors to bind to the apical membrane of WD cells is depicted on the left cell; the failure of “non-specifically” bound vectors to internalize is shown on the right cell. The tight junctions separate the apical cell membrane from the basolateral domain that selectively expresses specific viral receptors, e.g., the CAR, “housekeeping”/growth receptors, and integrins. WD, well-differentiated. CAR, Coxsackie virus and adenovirus receptor.
Figure 2
Figure 2
Strategies to increase adenovirus-mediated gene-transfer efficiency for well-differentiated columnar airway epithelial cells. (a) “Modification of the host.” In this approach, the tight junctions are rendered permeable to vectors, which permits access to “vector-specific” receptors. (b) “Modification of the vector.” A representative seven-transmembrane (7-TM) G-protein–receptor is shown that is activated to internalize via interaction with an agonist (denoted by purple-filled circle). Representative (adenovirus) vector is directed toward an external epitope of the receptor by bispecific antibodies (bs–Ab). Upon exposure to agonist, the 7-TM receptor is sequestered into a clathrin coated pit, carrying the vector into the cell.

Comment on

References

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