Advances in Duchenne muscular dystrophy gene therapy

被引：154

作者：

van Deutekom, JCT ^{[1
]}

van Ommen, GJB ^{[1
]}

机构：

[1] Leiden Univ, Med Ctr, Ctr Human & Clin Genet, NL-2333 AL Leiden, Netherlands

来源：

NATURE REVIEWS GENETICS | 2003年 / 4卷 / 10期

关键词：

D O I：

10.1038/nrg1180

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Since the initial characterization of the genetic defect for Duchenne muscular dystrophy, much effort has been expended in attempts to develop a therapy for this devastating childhood disease. Gene therapy was the obvious answer but, initially, the dystrophin gene and its product seemed too large and complex for this approach. However, our increasing knowledge of the organization of the gene and the role of dystrophin in muscle function has indicated ways to manipulate them both. Gene therapy for Duchenne muscular dystrophy now seems to be in reach.

引用

页码：774 / 783

页数：10

共 132 条

[61] 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

[62] Characterization of tissue tropism determinants of adeno-associated virus type 1 [J].

Hauck, B ;

Xiao, WD .

JOURNAL OF VIROLOGY, 2003, 77 (04) :2768-2774

[63] Exploiting the natural diversity in adenovirus tropism for therapy and prevention of disease [J].

Havenga, MJE ;

Lemckert, AAC ;

Ophorst, OJAE ;

van Meijer, M ;

Germeraad, WTV ;

Grimbergen, J ;

van den Doel, MA ;

Vogels, R ;

van Deutekom, J ;

Janson, AAM ;

de Bruijn, JD ;

Uytdehaag, F ;

Quax, PHA ;

Logtenberg, T ;

Mehtali, M ;

Bout, A .

JOURNAL OF VIROLOGY, 2002, 76 (09) :4612-4620

[64] Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus [J].

Herzog, RW ;

Hagstrom, JN ;

Kung, SH ;

Tai, SJ ;

Wilson, JM ;

Fisher, KJ ;

High, KA .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (11) :5804-5809

[65] A role for natural killer cells in the rapid death of cultured donor myoblasts after transplantation [J].

Hodgetts, SI ;

Spencer, MJ ;

Grounds, MD .

TRANSPLANTATION, 2003, 75 (06) :863-871

[66] DYSTROPHIN - THE PROTEIN PRODUCT OF THE DUCHENNE MUSCULAR-DYSTROPHY LOCUS [J].

HOFFMAN, EP ;

BROWN, RH ;

KUNKEL, LM .

CELL, 1987, 51 (06) :919-928

[67] Gene transfer to muscle using herpes simplex virus-based vectors [J].

Huard, J ;

Krisky, D ;

Oligino, T ;

Marconi, P ;

Day, CS ;

Watkins, SC ;

Glorioso, JC .

NEUROMUSCULAR DISORDERS, 1997, 7 (05) :299-313

[68] LOCALIZATION AND QUANTITATION OF THE CHROMOSOME 6-ENCODED DYSTROPHIN-RELATED PROTEIN IN NORMAL AND PATHOLOGICAL HUMAN MUSCLE [J].

KARPATI, G ;

CARPENTER, S ;

MORRIS, GE ;

DAVIES, KE ;

GUERIN, C ;

HOLLAND, P .

JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY, 1993, 52 (02) :119-128

[69] Activation of utrophin promoter by heregulin via the ets-related transcription factor complex GA-binding protein α/β [J].

Khurana, TS ;

Rosmarin, AG ;

Shang, J ;

Krag, TOB ;

Das, S ;

Gammeltoft, S .

MOLECULAR BIOLOGY OF THE CELL, 1999, 10 (06) :2075-2086

[70] A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase [J].

Kochanek, S ;

Clemens, PR ;

Mitani, K ;

Chen, HH ;

Chan, S ;

Caskey, CT .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1996, 93 (12) :5731-5736

← 2 3 4 5 6 7 8 9 10 11 →