Gene therapy of muscular dystrophy

被引：85

作者：

Chamberlain, JS ^{[1
]}

机构：

[1] Univ Washington, Sch Med, Dept Neurol, Seattle, WA 98195 USA

来源：

HUMAN MOLECULAR GENETICS | 2002年 / 11卷 / 20期

基金：

美国国家卫生研究院;

关键词：

D O I：

10.1093/hmg/11.20.2355

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：

Development of gene therapy for the muscular dystrophies represents a daunting challenge requiring significant advances in our knowledge of the defective genes, muscle promoters, viral vectors, immune system surveillance and methods for systemic delivery of vectors. However, tremendous progress has been made in developing improved viral vectors and avoiding immune reactions against gene transfer. This review summarizes recent progress and highlights problems that must be solved before an effective treatment is available.

引用

页码：2355 / 2362

页数：8

共 115 条

[41] Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector [J].

Greelish, JP ;

Su, LT ;

Lankford, EB ;

Burkman, JM ;

Chen, HY ;

Konig, SK ;

Mercier, IM ;

Desjardins, PR ;

Mitchell, MA ;

Zheng, XG ;

Leferovich, J ;

Gao, GP ;

Balice-Gordon, RJ ;

Wilson, JM ;

Stedman, HH .

NATURE MEDICINE, 1999, 5 (04) :439-443

[42] Combinatorial blockade of calcineurin and CD28 signaling facilitates primary and secondary therapeutic gene transfer by adenovirus vectors in dystrophic (mdx) mouse muscles [J].

Guibinga, GH ;

Lochmuller, H ;

Massie, B ;

Nalbantoglu, J ;

Karpati, G ;

Petrof, BJ .

JOURNAL OF VIROLOGY, 1998, 72 (06) :4601-4609

[43] Dystrophin expression in the mdx mouse restored by stem cell transplantation [J].

Gussoni, E ;

Soneoka, Y ;

Strickland, CD ;

Buzney, EA ;

Khan, MK ;

Flint, AF ;

Kunkel, LM ;

Mulligan, RC .

NATURE, 1999, 401 (6751) :390-394

[44] Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy [J].

Harper, SQ ;

Hauser, MA ;

DelloRusso, C ;

Duan, DS ;

Crawford, RW ;

Phelps, SF ;

Harper, HA ;

Robinson, AS ;

Engelhardt, JF ;

Brooks, SV ;

Chamberlain, JS .

NATURE MEDICINE, 2002, 8 (03) :253-261

[45]

Hartigan-O'Connor D, 2000, MICROSC RES TECHNIQ, V48, P223, DOI 10.1002/(SICI)1097-0029(20000201/15)48:3/4<223::AID-JEMT10>3.0.CO

[46]

2-L

[47]

Hartigan-O'Connor D, 2002, METHOD ENZYMOL, V346, P224

[48] Immune evasion by muscle-specific gene expression in dystrophic muscle [J].

Hartigan-O'Connor, D ;

Kirk, CJ ;

Crawford, R ;

Mulé, JJ ;

Chamberlain, JS .

MOLECULAR THERAPY, 2001, 4 (06) :525-533

[49] Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors [J].

Hauser, MA ;

Robinson, A ;

Hartigan-O'Connor, D ;

Williams-Gregory, D ;

Buskin, JN ;

Apone, S ;

Kirk, CJ ;

Hardy, S ;

Hauschka, SD ;

Chamberlain, JS .

MOLECULAR THERAPY, 2000, 2 (01) :16-25

[50] MUTATIONS IN THE LAMININ ALPHA-2-CHAIN GENE (LAMA2) CAUSE MEROSIN-DEFICIENT CONGENITAL MUSCULAR-DYSTROPHY [J].

HELBLINGLECLERC, A ;

ZHANG, X ;

TOPALOGLU, H ;

CRUAUD, C ;

TESSON, F ;

WEISSENBACH, J ;

TOME, FMS ;

SCHWARTZ, K ;

FARDEAU, M ;

TRYGGVASON, K ;

GUICHENEY, P .

NATURE GENETICS, 1995, 11 (02) :216-218

← 1 2 3 4 5 6 7 8 9 10 →