Response to Buratti et al.:: Can a 'patch' in a skipped exon make the pre-mRNA splicing machine run better?

被引：6

作者：

Eperon, IC ^{[1
]}

Muntoni, F

机构：

[1] Univ Leicester, Dept Biochem, Leicester LE1 7RH, Leics, England

[2] Univ London Imperial Coll Sci Technol & Med, Hammersmith Hosp, Dubowitz Neuromuscular Ctr, London W12 0NN, England

来源：

TRENDS IN MOLECULAR MEDICINE | 2003年 / 9卷 / 06期

关键词：

D O I：

10.1016/S1471-4914(03)00068-6

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：

In this issue, Buratti et A discuss a recent paper on the possibility of using complementary sequences to target RS (arginine- and serine-rich) domains to defective splicing enhancers. They raise an important issue: does the method work in cells? We have developed a similar method and have demonstrated that it does indeed rescue splicing of exon 7 of the SMN2 gene in fibroblasts derived from a patient.

引用

页码：233 / 234

页数：2

共 2 条

[1] Correction of disease-associated exon skipping by synthetic exon-specific activators [J].

Cartegni, L ;

Krainer, AR .

NATURE STRUCTURAL BIOLOGY, 2003, 10 (02) :120-125

[2] Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts [J].

Skordis, LA ;

Dunckley, MG ;

Yue, BG ;

Eperon, IC ;

Muntoni, F .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (07) :4114-4119

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