GENE-THERAPY AND PHARMACOLOGICAL TREATMENT OF INHERITED NEUROLOGICAL DISORDERS

被引:16
作者
MARTIN, JB
机构
[1] The Department of Neurology, University of California, San Francisco, CA 94143-0402
关键词
D O I
10.1016/S0167-7799(00)88898-8
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
The identification of mutant genes responsible for inherited neurological disorders gives an opportunity to consider new approaches to their treatment. Although replacement of defective genes in postmitotic neurons is unlikely to be possible in the near future, the identification of gene products, and definitive delineation of the cellular dysfunction and death that result from mutated gene products, may suggest new therapeutic options. This article reviews these advances and considers approaches that may provide hope for alleviation of these disorders.
引用
收藏
页码:28 / 35
页数:8
相关论文
共 45 条
[1]   HUMAN DYSTROPHIN EXPRESSION IN MDX MICE AFTER INTRAMUSCULAR INJECTION OF DNA CONSTRUCTS [J].
ACSADI, G ;
DICKSON, G ;
LOVE, DR ;
JANI, A ;
WALSH, FS ;
GURUSINGHE, A ;
WOLFF, JA ;
DAVIES, KE .
NATURE, 1991, 352 (6338) :815-818
[2]   HUMAN GENE-THERAPY [J].
ANDERSON, WF .
SCIENCE, 1992, 256 (5058) :808-813
[3]   THE RELATIONSHIP BETWEEN TRINUCLEOTIDE (CAG) REPEAT LENGTH AND CLINICAL-FEATURES OF HUNTINGTONS-DISEASE [J].
ANDREW, SE ;
GOLDBERG, YP ;
KREMER, B ;
TELENIUS, H ;
THEILMANN, J ;
ADAM, S ;
STARR, E ;
SQUITIERI, F ;
LIN, BY ;
KALCHMAN, MA ;
GRAHAM, RK ;
HAYDEN, MR .
NATURE GENETICS, 1993, 4 (04) :398-403
[4]   THE NF1 LOCUS ENCODES A PROTEIN FUNCTIONALLY RELATED TO MAMMALIAN GAP AND YEAST IRA PROTEINS [J].
BALLESTER, R ;
MARCHUK, D ;
BOGUSKI, M ;
SAULINO, A ;
LETCHER, R ;
WIGLER, M ;
COLLINS, F .
CELL, 1990, 63 (04) :851-859
[5]   THYMIDINE KINASE-MEDIATED KILLING OF RAT-BRAIN TUMORS [J].
BARBA, D ;
HARDIN, J ;
RAY, J ;
GAGE, FH .
JOURNAL OF NEUROSURGERY, 1993, 79 (05) :729-735
[6]   A CONTROLLED TRIAL OF RILUZOLE IN AMYOTROPHIC-LATERAL-SCLEROSIS [J].
BENSIMON, G ;
LACOMBLEZ, L ;
MEININGER, V ;
BOUCHE, P ;
DELWAIDE, C ;
COURATIER, P ;
BLIN, O ;
VIADER, F ;
PEYROSTPAUL, H ;
DAVID, J ;
MALOTEAUX, JM ;
HUGON, J ;
LATERRE, EC ;
RASCOL, A ;
CLANET, M ;
VALLAT, JM ;
DUMAS, A ;
SERRATRICE, G ;
LECHEVALLIER, B ;
PEUCH, AJ ;
NGUYEN, T ;
SHU, C ;
BASTIEN, P ;
PAPILLON, C ;
DURRLEMAN, S ;
LOUVEL, E ;
GUILLET, P ;
LEDOUX, L ;
ORVOENFRIJA, E ;
DIB, M .
NEW ENGLAND JOURNAL OF MEDICINE, 1994, 330 (09) :585-591
[7]  
CLEMENS PR, 1994, CURR NEUROL, V14, P29
[8]   PROTECTIVE EFFECT OF APOLIPOPROTEIN-E TYPE-2 ALLELE FOR LATE-ONSET ALZHEIMER-DISEASE [J].
CORDER, EH ;
SAUNDERS, AM ;
RISCH, NJ ;
STRITTMATTER, WJ ;
SCHMECHEL, DE ;
GASKELL, PC ;
RIMMLER, JB ;
LOCKE, PA ;
CONNEALLY, PM ;
SCHMADER, KE ;
SMALL, GW ;
ROSES, AD ;
HAINES, JL ;
PERICAKVANCE, MA .
NATURE GENETICS, 1994, 7 (02) :180-184
[9]   INVIVO GENE-TRANSFER WITH RETROVIRAL VECTOR PRODUCER CELLS FOR TREATMENT OF EXPERIMENTAL BRAIN-TUMORS [J].
CULVER, KW ;
RAM, Z ;
WALLBRIDGE, S ;
ISHII, H ;
OLDFIELD, EH ;
BLAESE, RM .
SCIENCE, 1992, 256 (5063) :1550-1552
[10]   DIRECT RETROVIRAL-MEDIATED TRANSFER OF A DYSTROPHIN MINIGENE INTO MDX MOUSE MUSCLE INVIVO [J].
DUNCKLEY, MG ;
WELLS, DJ ;
WALSH, FS ;
DICKSON, G .
HUMAN MOLECULAR GENETICS, 1993, 2 (06) :717-723