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Dystrophin immunity in Duchenne's muscular dystrophy. 2010
Jerry R Mendell, and
Katherine Campbell, and
Louise Rodino-Klapac, and
Zarife Sahenk, and
Chris Shilling, and
Sarah Lewis, and
Dawn Bowles, and
Steven Gray, and
Chengwen Li, and
Gloria Galloway, and
Vinod Malik, and
Brian Coley, and
K Reed Clark, and
Juan Li, and
Xiao Xiao, and
Jade Samulski, and
Scott W McPhee, and
R Jude Samulski, and
Christopher M Walker
Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
We report on delivery of a functional dystrophin transgene to skeletal muscle in six patients with Duchenne's muscular dystrophy. Dystrophin-specific T cells were detected after treatment, providing evidence of transgene expression even when the functional protein was not visualized in skeletal muscle. Circulating dystrophin-specific T cells were unexpectedly detected in two patients before vector treatment. Revertant dystrophin fibers, which expressed functional, truncated dystrophin from the deleted endogenous gene after spontaneous in-frame splicing, contained epitopes targeted by the autoreactive T cells. The potential for T-cell immunity to self and nonself dystrophin epitopes should be considered in designing and monitoring experimental therapies for this disease. (Funded by the Muscular Dystrophy Association and others; ClinicalTrials.gov number, NCT00428935.).
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Vnitrni lekarstvi,
