The application of ribozymes and DNAzymes in muscle and brain.
The application of ribozymes and DNAzymes in muscle and brain.
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DOI：
10.3390/molecules15085460
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发表时间：
2010-08-09
期刊：
影响因子：
--
通讯作者：
Phylactou LA
中科院分区：
文献类型：
--
作者：
Mastroyiannopoulos NP;Uney JB;Phylactou LA
催化核酸（CNA）的发现为科学家提供了有价值的工具，用于通过下调或调节参与这些疾病的内源性基因表达来鉴定几种未经治疗的疾病的新疗法。这些CNA旨在消除或修复病理性基因表达。核酶是一类CNA，主要用于下调（通过RNA切割）或修复（通过RNA反式剪接）疾病中涉及的不需要的基因表达。通过体外选择过程衍生的DNA酶也能够结合和切割RNA靶标，因此下调基因表达。本文综述了核酶和DNA酶在肌肉和脑中的应用。有几种疾病影响肌肉和大脑，催化核酸已被用作靶向这些疾病组中涉及的特定细胞转录物的工具。
The discovery of catalytic nucleic acids (CNAs) has provided scientists with valuable tools for the identification of new therapies for several untreated diseases through down regulation or modulation of endogenous gene expression involved in these ailments. These CNAs aim either towards the elimination or repair of pathological gene expression. Ribozymes, a class of CNAs, can be mostly used to down-regulate (by RNA cleavage) or repair (by RNA trans-splicing) unwanted gene expression involved in disease. DNAzymes, derived by in vitro selection processes are also able to bind and cleave RNA targets and therefore down-regulate gene expression. The purpose of this review is to present and discuss several applications of ribozymes and DNAzymes in muscle and brain. There are several diseases which affect muscle and brain and catalytic nucleic acids have been used as tools to target specific cellular transcripts involved in these groups of diseases.
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影响因子：
11.4
作者：
Kanamori, T;Nishimaki, K;Ohta, S
通讯作者：
Ohta, S
DOI：
10.1038/nrd2310
发表时间：
2007-06
期刊：
Nature reviews. Drug discovery
影响因子：
--
作者：
de Fougerolles A;Vornlocher HP;Maraganore J;Lieberman J
通讯作者：
Lieberman J
DOI：
10.1073/pnas.93.25.14753
发表时间：
1996-12-10
影响因子：
11.1
作者：
Czubayko, F;Schulte, AM;Wellstein, A
通讯作者：
Wellstein, A
影响因子：
4.8
作者：
Bowden, ET;Stoica, GE;Wellstein, A
通讯作者：
Wellstein, A
影响因子：
64.5
作者：
GUERRIERTAKADA, C;GARDINER, K;ALTMAN, S
通讯作者：
ALTMAN, S