标题
Gene-targeting pharmaceuticals for single-gene disorders
作者
关键词
-
出版物
HUMAN MOLECULAR GENETICS
Volume 25, Issue R1, Pages R18-R26
出版商
Oxford University Press (OUP)
发表日期
2015-12-31
DOI
10.1093/hmg/ddv476
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
- (2016) Li Xu et al. MOLECULAR THERAPY
- Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides
- (2015) Richard S. Geary et al. ADVANCED DRUG DELIVERY REVIEWS
- Exon skipping therapy for Duchenne muscular dystrophy
- (2015) Ryszard Kole et al. ADVANCED DRUG DELIVERY REVIEWS
- Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7
- (2015) John F. Staropoli et al. GENOMICS
- Progress and prospects of gene therapy clinical trials for the muscular dystrophies
- (2015) Niclas E. Bengtsson et al. HUMAN MOLECULAR GENETICS
- Expanding the Biologist’s Toolkit with CRISPR-Cas9
- (2015) Samuel H. Sternberg et al. MOLECULAR CELL
- Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
- (2015) Christoph Niemietz et al. MOLECULES
- Perspective: Enforce the clinical guidelines
- (2015) Monique Roobol NATURE
- Alnylam, Dicerna tussle over RNAi tech
- (2015) NATURE BIOTECHNOLOGY
- European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene
- (2015) Manuel Haas et al. NEUROMUSCULAR DISORDERS
- Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia
- (2015) Daniel Gaudet et al. NEW ENGLAND JOURNAL OF MEDICINE
- Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
- (2015) Nicole Ali McNeer et al. Nature Communications
- Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons
- (2015) Chul-Yong Park et al. Cell Reports
- Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs
- (2015) Amy L. Firth et al. Cell Reports
- Therapeutic potentials of gene silencing by RNA interference: Principles, challenges, and new strategies
- (2014) Yan Deng et al. GENE
- Programmable RNA recognition and cleavage by CRISPR/Cas9
- (2014) Mitchell R. O’Connell et al. NATURE
- Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
- (2014) Hao Yin et al. NATURE BIOTECHNOLOGY
- Targeting APOC3 in the Familial Chylomicronemia Syndrome
- (2014) Daniel Gaudet et al. NEW ENGLAND JOURNAL OF MEDICINE
- Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients
- (2014) Niels H. Skotte et al. PLoS One
- Antisense Oligonucleotides Capable of Promoting Specific Target mRNA Reduction via Competing RNase H1-Dependent and Independent Mechanisms
- (2014) Timothy A. Vickers et al. PLoS One
- Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial
- (2014) Eitan Kerem et al. Lancet Respiratory Medicine
- Eteplirsen for the treatment of Duchenne muscular dystrophy
- (2013) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Mipomersen, an Apolipoprotein B Synthesis Inhibitor, Reduces Atherogenic Lipoproteins in Patients With Severe Hypercholesterolemia at High Cardiovascular Risk
- (2013) Gregory S. Thomas et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
- (2013) Kevin Fitzgerald et al. LANCET
- Topoisomerases facilitate transcription of long genes linked to autism
- (2013) Ian F. King et al. NATURE
- Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS
- (2013) Michael E. Østergaard et al. NUCLEIC ACIDS RESEARCH
- Truncation of Ube3a-ATS Unsilences Paternal Ube3a and Ameliorates Behavioral Defects in the Angelman Syndrome Mouse Model
- (2013) Linyan Meng et al. PLoS Genetics
- Antisense-based therapy for the treatment of spinal muscular atrophy
- (2012) Frank Rigo et al. JOURNAL OF CELL BIOLOGY
- Polymer delivery systems for site-specific genome editing
- (2011) Nicole Ali McNeer et al. JOURNAL OF CONTROLLED RELEASE
- Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons
- (2011) Hsien-Sung Huang et al. NATURE
- Current prospects for RNA interference-based therapies
- (2011) Beverly L. Davidson et al. NATURE REVIEWS GENETICS
- RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform
- (2010) C. Frank Bennett et al. Annual Review of Pharmacology and Toxicology
- Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease
- (2010) Chiara Zuccato et al. PHYSIOLOGICAL REVIEWS
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