New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials

被引：123

作者：

Cossu, Giulio ^{[1
]}

Sampaolesi, Maurilio ^{[2
,3
]}

机构：

[1] Hosp San Raffaele, Stem Cell Res Inst, I-20132 Milan, Italy

[2] Univ Pavia, Dept Expt Med, Human Anat Sect, I-27100 Pavia, Italy

[3] Catholic Univ Louvain, Stem Cell Res Inst, B-3000 Louvain, Belgium

来源：

TRENDS IN MOLECULAR MEDICINE | 2007年 / 13卷 / 12期

关键词：

D O I：

10.1016/j.molmed.2007.10.003

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes, mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise patient mobility and quality of life, and in the most severe cases lead to complete paralysis and premature death. Although muscular dystrophies still lack an effective therapy, several novel strategies are entering or are ready to enter clinical trials. Here we review the main experimental strategies, namely drug, gene and cell therapies, outlining their goals and limitations. We also provide an update of ongoing or planned clinical trials based on these strategies.

引用

页码：520 / 526

页数：7

共 66 条

[1] Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients [J].

Aartsma-Rus, A ;

Janson, AAM ;

Kaman, WE ;

Bremmer-Bout, M ;

den Dunnen, JT ;

Baas, F ;

van Ommen, GJB ;

van Deutekom, JCT .

HUMAN MOLECULAR GENETICS, 2003, 12 (08) :907-914

[2] Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology [J].

Alter, J ;

Lou, F ;

Rabinowitz, A ;

Yin, HF ;

Rosenfeld, J ;

Wilton, SD ;

Partridge, TA ;

Lu, QL .

NATURE MEDICINE, 2006, 12 (02) :175-177

[3] Comparative analysis of antisense oligonucleotide sequences for targeted skipping of Exon 51 during dystrophin Pre-mRNA splicing in human muscle [J].

Arechavala-Gomeza, V. ;

Graham, I. R. ;

Popplewell, L. J. ;

Adams, A. M. ;

Aartsma-Rus, A. ;

Kinali, M. ;

Morgan, J. E. ;

Van Deutekom, J. C. ;

Wilton, S. D. ;

Dickson, G. ;

Muntoni, F. .

HUMAN GENE THERAPY, 2007, 18 (09) :798-810

[4] Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells [J].

Bachrach, E ;

Li, S ;

Perez, AL ;

Schienda, J ;

Liadaki, K ;

Volinski, J ;

Flint, A ;

Chamberlain, J ;

Kunkel, LM .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2004, 101 (10) :3581-3586

[5] Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice [J].

Barton, ER ;

Morris, L ;

Musaro, A ;

Rosenthal, N ;

Sweeney, HL .

JOURNAL OF CELL BIOLOGY, 2002, 157 (01) :137-147

[6] Multipotential mesoangioblast stem cell therapy in the mdx/utrn-/- mouse model for Duchenne muscular dystrophy [J].

Berry, Suzanne E. ;

Liu, Jianming ;

Chaney, Eric J. ;

Kaufman, Stephen J. .

REGENERATIVE MEDICINE, 2007, 2 (03) :275-288

[7] Function and genetics of dystrophin and dystrophin-related proteins in muscle [J].

Blake, DJ ;

Weir, A ;

Newey, SE ;

Davies, KE .

PHYSIOLOGICAL REVIEWS, 2002, 82 (02) :291-329

[8] Functional improvement of dystrophic muscle by myostatin blockade [J].

Bogdanovich, S ;

Krag, TOB ;

Barton, ER ;

Morris, LD ;

Whittemore, LA ;

Ahima, RS ;

Khurana, TS .

NATURE, 2002, 420 (6914) :418-421

[9] Targeted exon skipping in transgenic hDMD mice:: A model for direct preclinical screening of human-specific antisense oligonucleotides [J].

Bremmer-Bout, M ;

Aartsma-Rus, A ;

de Meijer, EJ ;

Kaman, WE ;

Janson, AAM ;

Vossen, RHAM ;

van Ommen, GJB ;

den Dunnen, JT ;

van Deutekom, JCT .

MOLECULAR THERAPY, 2004, 10 (02) :232-240

[10] Too much hype, not enough hope: Are balanced reporting and proper controls too much to expect from therapeutic studies in animal models of neuromuscular diseases that presage clinical trials in humans? [J].

Bretag, Allan .

NEUROMUSCULAR DISORDERS, 2007, 17 (03) :203-205

← 1 2 3 4 5 6 7 →