TY - JOUR
AU - Fardeau, Michel
AB - Nine patients with Duchenne or Becker muscular dystrophy were injected via the radialis muscle with a full-length human dystrophin plasmid, either once with 200 or 600 microg of DNA or twice, 2 weeks apart, with 600 microg of DNA. In the biopsies taken 3 weeks after the initial injection, the vector was detected at the injection site in all patients. Immunohistochemistry and nested reverse transcription-polymerase chain reaction indicated dystrophin expression in six of nine patients. The level of expression was low (up to 6% weak, but complete sarcolemmal dystrophin staining, and up to 26% partial sarcolemmal labeling). No side effects were observed, nor any cellular or humoral anti-dystrophin responses. These results suggest that exogenous dystrophin expression can be obtained in Duchenne/Becker patients after intramuscular transfer of plasmid, without adverse effects, hence paving the way for future developments in gene therapy of hereditary muscular diseases.
TI - Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy.
JF - Human gene therapy
DO - 10.1089/hum.2004.15.1065
DA - 2005-05-05
UR - https://www.deepdyve.com/lp/pubmed/phase-i-study-of-dystrophin-plasmid-based-gene-therapy-in-duchenne-BU3DM0NYj1
SP - 1065
EP - 76
VL - 15
IS - 11
DP - DeepDyve
ER -