Therapeutic strategies for the inherited neuropathies

被引：28

作者：

Shy, Michael E. ^{[1
]}

机构：

[1] Wayne State Univ, Sch Med, Dept Neurol, Detroit, MI 48201 USA

[2] Wayne State Univ, Sch Med, Ctr Mol Med & Genet, Detroit, MI 48201 USA

来源：

NEUROMOLECULAR MEDICINE | 2006年 / 8卷 / 1-2期

关键词：

charcot-Marie-Tooth disease; neuropathy; genetics; therapy; disease mechanisms; gene therapy;

D O I：

10.1385/NMM:8:1:255

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

More than 30 genetic causes have been identified for the inherited neuropathies collectively referred to as Charcot-Marie-Tooth (CMT) disease. Previous therapies for CMT were limited to traditional approaches such as rehabilitation medicine, ambulation aids, and pain management. Identification of the genes causing CMT has led to improved genetic counseling and assistance in family planning. Identification of these genes is beginning to delineate common molecular pathways in multiple forms of CMT that can be exploited in future molecular therapies. Scientifically based clinical trials for CMT are currently being implemented. Techniques of gene therapy are advancing to the point that they may become feasible options for patients with CMT and other neurodegenerative diseases.

引用

页码：255 / 278

页数：24

共 148 条

[1] Increased survival and function of SOD1 mice after glial cell-derived neurotrophic factor gene therapy [J].

Acsadi, G ;

Anguelov, RA ;

Yang, HB ;

Toth, G ;

Thomas, R ;

Jani, A ;

Wang, YY ;

Ianakova, E ;

Mohammad, S ;

Lewis, RA ;

Shy, ME .

HUMAN GENE THERAPY, 2002, 13 (09) :1047-1059

[2]

ACSADI G, 1995, J MOL MED, V73, P165

[3]

[Anonymous], 1886, PERONEAL TYPE PROGRE

[4] Glycyl tRNA synthetase mutations in Charcot-Marie-Tooth disease type 2D and distal spinal muscular atrophy type V [J].

Antonellis, A ;

Ellsworth, RE ;

Sambuughin, N ;

Puls, I ;

Abel, A ;

Lee-Lin, SQ ;

Jordanova, A ;

Kremensky, I ;

Christodoulou, K ;

Middleton, LT ;

Sivakumar, K ;

Ionasescu, V ;

Funalot, B ;

Vance, JM ;

Goldfarb, LG ;

Fischbeck, KH ;

Green, ED .

AMERICAN JOURNAL OF HUMAN GENETICS, 2003, 72 (05) :1293-1299

[5]

Apfel SC, 2002, INT REV NEUROBIOL, V50, P393

[6] Is the therapeutic application of neurotrophic factors dead? [J].

Apfel, SC .

ANNALS OF NEUROLOGY, 2002, 51 (01) :8-11

[7] On the molecular architecture of myelinated fibers [J].

Arroyo, EJ ;

Scherer, SS .

HISTOCHEMISTRY AND CELL BIOLOGY, 2000, 113 (01) :1-18

[8] Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model [J].

Arruda, VR ;

Stedman, HH ;

Nichols, TC ;

Haskins, ME ;

Nicholson, M ;

Herzog, RW ;

Couto, LB ;

High, KA .

BLOOD, 2005, 105 (09) :3458-3464

[9] VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model [J].

Azzouz, M ;

Ralph, GS ;

Storkebaum, E ;

Walmsley, LE ;

Mitrophanous, KA ;

Kingsman, SM ;

Carmeliet, P ;

Mazarakis, ND .

NATURE, 2004, 429 (6990) :413-417

[10] Back signaling by the Nrg-1 intracellular domain [J].

Bao, JX ;

Wolpowitz, D ;

Role, LW ;

Talmage, DA .

JOURNAL OF CELL BIOLOGY, 2003, 161 (06) :1133-1141

← 1 2 3 4 5 6 7 8 9 10 →