Correction of genetic disease by making sense from nonsense

被引：42

作者：

Kaufman, RJ ^{[1
]}

机构：

[1] Univ Michigan, Sch Med, Howard Hughes Med Inst, Ann Arbor, MI 48109 USA

来源：

JOURNAL OF CLINICAL INVESTIGATION | 1999年 / 104卷 / 04期

关键词：

D O I：

10.1172/JCI8055

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

引用

页码：367 / 368

页数：2

共 23 条

[1] Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice [J].

Barton-Davis, ER ;

Cordier, L ;

Shoturma, DI ;

Leland, SE ;

Sweeney, HL .

JOURNAL OF CLINICAL INVESTIGATION, 1999, 104 (04) :375-381

[2] Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line [J].

Bedwell, DM ;

Kaenjak, A ;

Benos, DJ ;

Bebok, Z ;

Bubien, JK ;

Hong, J ;

Tousson, A ;

Clancy, JP ;

Sorscher, EJ .

NATURE MEDICINE, 1997, 3 (11) :1280-1284

[3] EVIDENCE TO IMPLICATE TRANSLATION BY RIBOSOMES IN THE MECHANISM BY WHICH NONSENSE CODONS REDUCE THE NUCLEAR-LEVEL OF HUMAN TRIOSEPHOSPHATE ISOMERASE MESSENGER-RNA [J].

BELGRADER, P ;

CHENG, J ;

MAQUAT, LE .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1993, 90 (02) :482-486

[4] SUPPRESSION OF A NONSENSE MUTATION IN MAMMALIAN-CELLS INVIVO BY THE AMINOGLYCOSIDE ANTIBIOTICS G-418 AND PAROMOMYCIN [J].

BURKE, JF ;

MOGG, AE .

NUCLEIC ACIDS RESEARCH, 1985, 13 (17) :6265-6272

[5] Infrequent translation of a nonsense codon is sufficient to decrease mRNA level [J].

Buzina, A ;

Shulman, MJ .

MOLECULAR BIOLOGY OF THE CELL, 1999, 10 (03) :515-524

[6] EXPRESSION OF THE MURINE DUCHENNE MUSCULAR-DYSTROPHY GENE IN MUSCLE AND BRAIN [J].

CHAMBERLAIN, JS ;

PEARLMAN, JA ;

MUZNY, DM ;

GIBBS, RA ;

RANIER, JE ;

REEVES, AA ;

CASKEY, CT .

SCIENCE, 1988, 239 (4846) :1416-1418

[7] OVEREXPRESSION OF DYSTROPHIN IN TRANSGENIC MDX MICE ELIMINATES DYSTROPHIC SYMPTOMS WITHOUT TOXICITY [J].

COX, GA ;

COLE, NM ;

MATSUMURA, K ;

PHELPS, SF ;

HAUSCHKA, SD ;

CAMPBELL, KP ;

FAULKNER, JA ;

CHAMBERLAIN, JS .

NATURE, 1993, 364 (6439) :725-729

[8] Binding of neomycin-class aminoglycoside antibiotics to the A-site of 16 S rRNA [J].

Fourmy, D ;

Recht, MI ;

Puglisi, JD .

JOURNAL OF MOLECULAR BIOLOGY, 1998, 277 (02) :347-362

[9] Progress towards gene therapy for Duchenne muscular dystrophy [J].

Hauser, MA ;

Chamberlain, JS .

JOURNAL OF ENDOCRINOLOGY, 1996, 149 (03) :373-378

[10] Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations [J].

Howard, M ;

Frizzell, DM ;

Bedwell, DM .

NATURE MEDICINE, 1996, 2 (04) :467-469

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