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N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228.

Dystrophin immunity in Duchenne's muscular dystrophy.

Author information

1
Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.

Abstract

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.).

PMID:
20925545
PMCID:
PMC3014106
DOI:
10.1056/NEJMoa1000228
[Indexed for MEDLINE]
Free PMC Article

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