Skipping toward personalized molecular medicine

被引:46
作者
Hoffman, Eric P. [1 ]
机构
[1] George Washington Univ, Sch Med, Dept Pediat, Washington, DC 20052 USA
[2] George Washington Univ, Sch Med, Dept Neurosci, Washington, DC USA
[3] George Washington Univ, Sch Med, Dept Biochem & Mol Biol, Washington, DC USA
[4] Childrens Natl Med Ctr, Med Genet Res Ctr, Washington, DC 20010 USA
关键词
D O I
10.1056/NEJMe0707795
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:2719 / 2722
页数:4
相关论文
共 9 条
[1]   Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology [J].
Alter, J ;
Lou, F ;
Rabinowitz, A ;
Yin, HF ;
Rosenfeld, J ;
Wilton, SD ;
Partridge, TA ;
Lu, QL .
NATURE MEDICINE, 2006, 12 (02) :175-177
[2]   Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63 % of patients with Duchenne muscular dystrophy [J].
Beroud, Christophe ;
Tuffery-Giraud, Sylvie ;
Matsuo, Masafumi ;
Hamroun, Dalil ;
Humbertclaude, Wronique ;
Monnier, Nicole ;
Moizard, Marie-Pierre ;
Voelckel, Marie-Antoinette ;
Calemard, Laurence Michel ;
Boisseau, Pierre ;
Blayau, Martine ;
Philippe, Christophe ;
Cossee, Mireille ;
Pages, Michel ;
Rivier, Franois ;
Danos, Olivier ;
Garcia, Luis ;
Claustres, Mireille .
HUMAN MUTATION, 2007, 28 (02) :196-202
[3]   VERY MILD MUSCULAR-DYSTROPHY ASSOCIATED WITH THE DELETION OF 46-PERCENT OF DYSTROPHIN [J].
ENGLAND, SB ;
NICHOLSON, LVB ;
JOHNSON, MA ;
FORREST, SM ;
LOVE, DR ;
ZUBRZYCKAGAARN, EE ;
BULMAN, DE ;
HARRIS, JB ;
DAVIES, KE .
NATURE, 1990, 343 (6254) :180-182
[4]   DYSTROPHIN - THE PROTEIN PRODUCT OF THE DUCHENNE MUSCULAR-DYSTROPHY LOCUS [J].
HOFFMAN, EP ;
BROWN, RH ;
KUNKEL, LM .
CELL, 1987, 51 (06) :919-928
[5]   Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles [J].
Lu, QL ;
Rabinowitz, A ;
Chen, YC ;
Yokota, T ;
Yin, HF ;
Alter, J ;
Jadoon, A ;
Bou-Gharios, G ;
Partridge, T .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2005, 102 (01) :198-203
[6]  
Muntoni Francesco, 2005, Neuromuscul Disord, V15, P450, DOI 10.1016/j.nmd.2005.02.007
[7]   Local dystrophin restoration with antisense oligonucleotide PRO051 [J].
van Deutekom, Judith C. ;
Janson, Anneke A. ;
Ginjaar, Ieke B. ;
Frankhuizen, Wendy S. ;
Aartsma-Rus, Annemieke ;
Bremmer-Bout, Mattie ;
den Dunnen, Johan T. ;
Koop, Klaas ;
van der Kooi, Anneke J. ;
Goemans, Nathalie M. ;
de Kimpe, Sjef J. ;
Ekhart, Peter F. ;
Venneker, Edna H. ;
Platenburg, Gerard J. ;
Verschuuren, Jan J. ;
van Ommen, Gert-Jan B. .
NEW ENGLAND JOURNAL OF MEDICINE, 2007, 357 (26) :2677-2686
[8]  
YOKOTA T, IN PRESS ACTA MYOL
[9]   Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy [J].
Yokota, Toshifumi ;
Pistilli, Emidio ;
Duddy, William ;
Nagaraju, Kanneboyina .
EXPERT OPINION ON BIOLOGICAL THERAPY, 2007, 7 (06) :831-842