Towards gene therapy for the central nervous system

被引：25

作者：

During, MJ

Ashenden, LMA

机构：

[1] Thomas Jefferson Univ, Jefferson Med Coll, Jefferson CNS Gene Therapy, Dept Neurosurg, Philadelphia, PA 19107 USA

[2] Univ Auckland, Sch Med, Auckland, New Zealand

来源：

MOLECULAR MEDICINE TODAY | 1998年 / 4卷 / 11期

关键词：

D O I：

10.1016/S1357-4310(98)01370-7

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：

Gene therapy has generated enormous scientific, medical and public interest over the last decade. Clinical trials involving approximately 2000 patients worldwide have targeted simple genetic diseases such as cystic fibrosis, muscular dystrophy, adenosine deaminase deficiency, Gaucher's disease and familial hypercholesterolemia, as well as complex acquired diseases such as cancer and AIDS. The central nervous system is a new and particularly exciting target for gene therapy because its unique properties prevent the successful treatment of many neurological disorders by conventional means. This review discusses the potential applications of in vivo gene therapy to neurological disorders that have the greatest potential for genetic treatments.

引用

页码：485 / 493

页数：9

共 38 条

[1] The application of genetically engineered herpes simplex viruses to the treatment of experimental brain tumors [J].

Andreansky, SS ;

He, B ;

Gillespie, GY ;

Soroceanu, L ;

Markert, J ;

Chou, J ;

Roizman, B ;

Whitley, RJ .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1996, 93 (21) :11313-11318

[2] Intrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioral impairment in a rat model of Parkinson disease [J].

BilangBleuel, A ;

Revah, F ;

Colin, P ;

Locquet, I ;

Robert, JJ ;

Mallet, J ;

Horellou, P .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (16) :8818-8823

[3] Studies on neuroprotective and regenerative effects of GDNF in a partial lesion model of Parkinson's disease [J].

Bjorklund, A ;

Rosenblad, C ;

Winkler, C ;

Kirik, D .

NEUROBIOLOGY OF DISEASE, 1997, 4 (3-4) :186-200

[4] Bcl-xL protects adult septal cholinergic neurons from axotomized cell death [J].

Blömer, U ;

Kafri, T ;

Randolph-Moore, L ;

Verma, IM ;

Gage, FH .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1998, 95 (05) :2603-2608

[5] Dopaminergic neurons protected from degeneration by GDNF gene therapy [J].

ChoiLundberg, DL ;

Lin, Q ;

Chang, YN ;

Chiang, YL ;

Hay, CM ;

Mohajeri, H ;

Davidson, BL ;

Bohn, MC .

SCIENCE, 1997, 275 (5301) :838-841

[6] EXPRESSION OF ESCHERICHIA-COLI BETA-GALACTOSIDASE AND RAT HPRT IN THE CNS OF MACACA-MULATTA FOLLOWING ADENOVIRAL MEDIATED GENE-TRANSFER [J].

DAVIDSON, BL ;

DORAN, SE ;

SHEWACH, DS ;

LATTA, JM ;

HARTMAN, JW ;

ROESSLER, BJ .

EXPERIMENTAL NEUROLOGY, 1994, 125 (02) :258-267

[7]

DELIGANIS A, 1996, AM J NEURORADIOL, V18, P1401

[8] GENE-EXPRESSION FROM RECOMBINANT VIRAL VECTORS IN THE CENTRAL-NERVOUS-SYSTEM AFTER BLOOD-BRAIN-BARRIER DISRUPTION [J].

DORAN, SE ;

REN, XD ;

BETZ, AL ;

PAGEL, MA ;

NEUWELT, EA ;

ROESSLER, BJ ;

DAVIDSON, BL .

NEUROSURGERY, 1995, 36 (05) :965-970

[9]

DURING M, 1996, SOC NEUROSCI, V22

[10] Targets for gene therapy of Parkinson's disease: Growth factors, signal transduction, and promoters [J].

During, MJ ;

Leone, P .

EXPERIMENTAL NEUROLOGY, 1997, 144 (01) :74-81

← 1 2 3 4 →