Myotonic dystrophy: Antisense oligonucleotide inhibition of DMPK gene expression in vitro

被引：17

作者：

Galderisi, U ^{[1
]}

Cipollaro, M ^{[1
]}

Melone, MAB ^{[1
]}

Iacomino, G ^{[1
]}

DiBernardo, G ^{[1
]}

Galano, G ^{[1
]}

Contrufo, R ^{[1
]}

Zappia, V ^{[1
]}

Cascino, A ^{[1
]}

机构：

[1] UNIV NAPLES 2,FAC MED,DIV NEUROL 2,I-80138 NAPLES,ITALY

来源：

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS | 1996年 / 221卷 / 03期

关键词：

D O I：

10.1006/bbrc.1996.0668

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：

Antisense phosphorothioate oligonucleotides, targeted against the first codon starting region of DMPK mRNA, were successfully used in K562 and HepG2 cells to decrease DMPK expression. The most effective antisense oligo, MIO1, when added to K562 cells, shows a 75% reduction of the DMPK gene expression 6 hours after addition. The same molecule, when encapsulated in liposomes, delays myotonin mRNA decrease at 24 hours after cell treatment. This considerable success with such inhibition in vitro could be utilised to generate a cell model to study myotonic dystrophy (DM) chemio-physiological alterations. (C) 1996 Academic Press, Inc.

引用

页码：750 / 754

页数：5

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