相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Spinal muscular atrophy approval boosts antisense drugs
Elie Dolgin
NATURE BIOTECHNOLOGY (2017)
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
David R. Corey
NATURE NEUROSCIENCE (2017)
MicroRNA therapeutics: towards a new era for the management of cancer and other diseases
Rajesha Rupaimoole et al.
NATURE REVIEWS DRUG DISCOVERY (2017)
Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts
Sebastien A. Burel et al.
NUCLEIC ACIDS RESEARCH (2016)
Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience
Manon Hache et al.
JOURNAL OF CHILD NEUROLOGY (2016)
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Richard S. Finkel et al.
LANCET (2016)
Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy
Claudia A. Chiriboga et al.
NEUROLOGY (2016)
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
Suzan M. Hammond et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)
Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
Bailey Miskew Nichols et al.
JOVE-JOURNAL OF VISUALIZED EXPERIMENTS (2016)
Design and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro
Takenori Shimo et al.
NUCLEIC ACIDS RESEARCH (2014)
Evaluation of Multiple-Turnover Capability of Locked Nucleic Acid Antisense Oligonucleotides in Cell-Free RNase H-Mediated Antisense Reaction and in Mice
Tsuyoshi Yamamoto et al.
NUCLEIC ACID THERAPEUTICS (2014)
A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy
Jeffrey M. Keil et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2014)
A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice
Haiyan Zhou et al.
HUMAN GENE THERAPY (2013)
Splice Modulating Therapies for Human Disease
Pietro Spitali et al.
CELL (2012)
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
Paul N. Porensky et al.
HUMAN MOLECULAR GENETICS (2012)
Therapeutic inhibition of the miR-34 family attenuates pathological cardiac remodeling and improves heart function
Bianca C. Bernardo et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)
Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
Yimin Hua et al.
NATURE (2011)
Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
Marco A. Passini et al.
SCIENCE TRANSLATIONAL MEDICINE (2011)
Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
Yimin Hua et al.
GENES & DEVELOPMENT (2010)
An antisense microwalk reveals critical role of an intronic position linked to a unique long-distance interaction in pre-mRNA splicing
Natalia N. Singh et al.
RNA (2010)
Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection
Robert E. Lanford et al.
SCIENCE (2010)
A Positive Modifier of Spinal Muscular Atrophy in the SMN2 Gene
Thomas W. Prior et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2009)
A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy
Natalia N. Singh et al.
RNA BIOLOGY (2009)
Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in Transgenic mice
Yimin Hua et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2008)
Enhancement of SMN2 Exon 7 inclusion by antisense oligonucleotides targeting the exon
Yimin Hua et al.
PLOS BIOLOGY (2007)
Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice
Jennifer Roberts et al.
MOLECULAR THERAPY (2006)
Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron
NK Singh et al.
MOLECULAR AND CELLULAR BIOLOGY (2006)
Exploring cellular activity of locked nucleic acid-modified triplex-forming oligonucleotides and defining its molecular basis
E Brunet et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2005)
Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality
J Elmen et al.
NUCLEIC ACIDS RESEARCH (2005)
Potent and nontoxic antisense oligonucleotides containing locked nucleic acids
C Wahlestedt et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)