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Duchenne and Becker muscular dystrophy are caused by defects in the dystrophin gene, and are candidates for treatment by gene therapy. We have shown previously that overexpression of a full-length dystrophin cDNA prevents the development of dystrophic symptoms in mdx mice. We show here that this functional correction can be achieved by expressing the full-length muscle isoform at a lower level than is present in control animals. Gene therapy for DMD may necessitate the use of truncated dystrophin mini-genes to accommodate the limited cloning capacity of current-generation viral delivery vectors. We have constructed both murine and human mini-genes deleted for exons 17-48, and have demonstrated that expression of either mini-gene can almost completely prevent the development of dystrophic symptoms in transgenic mdx mice. These results suggest that viral-mediated expression of moderate levels of a truncated dystrophin could be an effective treatment for DMD.
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
October 1994,
Molecular biology of the cell,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
June 2004,
Gene therapy,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
June 2004,
Gene therapy,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
November 2006,
Molecular therapy : the journal of the American Society of Gene Therapy,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
February 2004,
The Journal of physiology,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
April 1992,
Human molecular genetics,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
June 2021,
FASEB journal : official publication of the Federation of American Societies for Experimental Biology,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
August 2004,
Human molecular genetics,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
September 2010,
Gene therapy,
S F Phelps, and
M A Hauser, and
N M Cole, and
J A Rafael, and
R T Hinkle, and
J A Faulkner, and
J S Chamberlain
February 2003,
Muscle & nerve,
