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Functional repair of a mutant chloride channel using a trans-splicing ribozyme
Christopher S. Rogers, … , Bruce A. Sullenger, Alfred L. George Jr.
Christopher S. Rogers, … , Bruce A. Sullenger, Alfred L. George Jr.
Published December 15, 2002
Citation Information: J Clin Invest. 2002;110(12):1783-1789. https://doi.org/10.1172/JCI16481.
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Article Genetics

Functional repair of a mutant chloride channel using a trans-splicing ribozyme

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Abstract

Research Article

Authors

Christopher S. Rogers, Carlos G. Vanoye, Bruce A. Sullenger, Alfred L. George Jr.

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Figure 5

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Spectrum of ribozyme-mediated restoration of cClC-1 function. (a) Voltag...
Spectrum of ribozyme-mediated restoration of cClC-1 function. (a) Voltage dependence of activation for wild-type cClC-1 (squares), T268M (diamonds), and three representative ribozyme-transfected T268M-stable cells (black lines). Dotted curves represent two SDs from the mean of T268M. (b) Scatter diagram illustrating the V1/2 determined for cells expressing wild-type cClC-1, T268M, and ribozyme-treated T268M. Data for the 13 cells demonstrating a significant level of functional repair are shown as individual data points (filled circles). Mean values for wild-type cClC-1 (square, n = 12), untreated T268M (diamond, n = 52), ribozyme-treated T268M exhibiting no repair (open circle, n = 58) and inactive ribozyme–treated T268M (triangle, n = 11) are shown as single points. Dotted line represents two SDs from the mean of T268M. Error bars represent SEM. (In some cases, the data symbols are larger than the error bars.)

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