Regulating Export of ER Cargo

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Science  04 Feb 2000:
Vol. 287, Issue 5454, pp. 816-817
DOI: 10.1126/science.287.5454.816

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One of the goals of gene therapy is the administration of genes (or their protein products) so that the therapeutic proteins are rapidly delivered to the correct location in a physiological manner. In a Perspective, Aridor and Balch discuss new findings ( Rivera et al. ) that capitalize on the ability of the endoplasmic reticulum (ER) to harbor protein aggregates for regulated delivery to the cell surface. The new work demonstrates that aggregates of a fusion protein (FKBP12 and either insulin or growth hormone) accumulate in the ER. Addition of a small-molecule drug that "dissolves" the aggregates results in rapid export of these therapeutic hormones and their release into the circulation.