自引率: 7.2%
被引量: 5024
通过率: 暂无数据
审稿周期: 4.75
版面费用: 暂无数据
国人发稿量: 41
期刊描述简介:
Molecular Therapy — Nucleic Acids is an international, open-access journal publishing top-quality basic, translational, and clinical research in the broad fields of nucleic acid-based therapeutics to treat and/or correct genetic and acquired disease. Subject areas include but are not limited to: development of therapeutics based on nucleic acids and their derivatives, vector development and design for the delivery of RNA-based therapeutics, applications of gene-modifying agents including triplex-forming oligonucleotides and enzymes such as Zn finger nucleases, pre-clinical target validation, safety/efficacy studies, and clinical trials. Molecular Therapy — Nucleic Acids 2015 impact factor is 5.048.
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Cas9 editing of ATXN1 in a spinocerebellar ataxia type 1 mice and human iPSC-derived neurons.
被引量:- 发表:1970
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Retraction Notice to: LncRNA PVT1 promotes tumorigenesis of glioblastoma by recruiting COPS5 to deubiquitinate and stabilize TRIM24.
被引量:- 发表:1970
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Targeting heterozygous dominant negative variant of KCNA2 using Gapmer ASO for the treatment of drug-resistant epilepsy.
被引量:- 发表:1970
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Simple improvements in vector design afford substantial gains in AAV delivery of aggregation-slowing Aβ variants.
被引量:- 发表:1970
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The role of RNA modifications in disease-associated macrophages.
被引量:- 发表:1970