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2014-10-17 - Colloque/Présentation - poster - Anglais - 1 page(s)

Derenne Aline, Tassin Alexandra , Conotte Stéphanie, Coppée Frédérique , Belayew Alexandra , Legrand Alexandre , "Comparison of two methods of naked DNA delivery in mice: which one is the most appropriate in the FSHD context?" in FSH Society international research consortium and research planning meeting, San Diego, USA, 2014

  • Codes CREF : Biologie moléculaire (DI3111)
  • Unités de recherche UMONS : Physiologie et réadaptation respiratoire (M117), Biochimie métabolique et moléculaire (M122)
  • Instituts UMONS : Institut des Sciences et Technologies de la Santé (Santé)

Abstract(s) :

(Anglais) expression in fibers localized close to the injection site, while the IVHD caused expression in scattered fibers among the different lower limb muscles. In conclusion, IVHD and IMEP naked DNA delivery yield different transgene expression levels and patterns which could both be useful in the context of an FSHD model. Indeed, the IMEP leads to a strong and local transgene expression which could allow an easier scoring of AO-mediated DUX4 suppression. The IVHD results in a pattern expected in FSHD patient muscles based on DUX4 stochastic expression observed in primary myoblast and myotube cultures. The IVHD model will thus be preferred for an investigation of pathophysiological mechanisms induced by DUX4 expression. References (1) Vanderplanck et al, PLoS One. 2011; 6(10):e26820. (2) Krom et al, PLoS Genet. 2013; 9(4):e1003415. (3) Dandapat et al Cell Rep. 2014; 11;8(5):1484-96 (4) Wallace et al, Ann Neurol. 2011; 69(3):540-52. (5) Hagstrom et al, Mol Ther. 2004; 10(2):386-98 (6) Wooddell et al, Hum Gene Ther. 2011; 22(7):889–903. (7) Vilquin et al, Gene Ther. 2001; 8(14):1097–107.