L. Vitiello et al., GENE-THERAPY FOR DUCHENNE MUSCULAR-DYSTROPHY - EARLY EXPERIENCES WITHLIPOSOME-MEDIATED GENE-TRANSFER, Transfusion science, 17(1), 1996, pp. 63-69
Duchenne muscular dystrophy is a lethal X-linked disorder in which mus
cle degeneration results in confinement to a wheelchair by age 12 year
s and death from respiratory failure by age 20 years. Gene therapy for
this disorder poses unique challenges, related to the enormous size o
f the gene, and to the difficulty of delivering a functional gene to t
he millions of muscle fibers through-out the body. Adenoviral vectors
are limited in their DNA capacity precluding their use to deliver an i
ntact dystrophin gene cDNA, and the vectors themselves elicit an immun
e response that will further limit their use. Cationic liposomes provi
de a means to transfect cells bother viva and in vivo. In this study w
e evaluated the use of a polycationic liposome formulation in deliveri
ng are porter gene in cultured muscle cells and designed a new procedu
re to enhance transfection efficiency of cationic liposomes, based on
the precondensation of plasmid DNA with polylysine.