Assisted delivery of antisense therapeutics in animal models of heritable neurodegenerative and neuromuscular disorders: a systematic review and meta-analysis

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

(2017) Lindsay A. Becker et al.   NATURE

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

(2017) Daniel R. Scoles et al.   NATURE

Oligonucleotide therapies for disorders of the nervous system

(2017) Olga Khorkova et al.   NATURE BIOTECHNOLOGY

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

(2017) Lodewijk J.A. Toonen et al.   Molecular Therapy-Nucleic Acids

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

(2017) Lauren R. Moore et al.   Molecular Therapy-Nucleic Acids

Approving a Problematic Muscular Dystrophy Drug

(2016) Aaron S. Kesselheim et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Intrathecal Injections in Children With Spinal Muscular Atrophy

(2016) Manon Haché et al.   JOURNAL OF CHILD NEUROLOGY

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

Delivery of oligonucleotides with lipid nanoparticles

(2015) Yuhua Wang et al.   ADVANCED DRUG DELIVERY REVIEWS

Delivery of therapeutic oligonucleotides with cell penetrating peptides

(2015) Prisca Boisguérin et al.   ADVANCED DRUG DELIVERY REVIEWS

Antisense oligonucleotides in therapy for neurodegenerative disorders

(2015) Melvin M. Evers et al.   ADVANCED DRUG DELIVERY REVIEWS

An update on RNA-targeting therapies for neuromuscular disorders

(2015) Silvana Jirka et al.   CURRENT OPINION IN NEUROLOGY

Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy

(2015) Kentaro Sahashi et al.   HUMAN MOLECULAR GENETICS

Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides

(2015) Umar Burki et al.   Nucleic Acid Therapeutics

SYRCLE’s risk of bias tool for animal studies

(2014) Carlijn R Hooijmans et al.   BMC Medical Research Methodology

Effect of Combined Systemic and Local Morpholino Treatment on the Spinal Muscular Atrophy Δ7 Mouse Model Phenotype

(2014) Monica Nizzardo et al.   CLINICAL THERAPEUTICS

Evaluation of Tris[2-(Acryloyloxy)Ethyl]Isocyanurate Cross-Linked Polyethylenimine as Antisense Morpholino Oligomer Delivery Vehicle in Cell Culture and Dystrophic mdx Mice

(2014) Mingxing Wang et al.   HUMAN GENE THERAPY

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Dystrophin quantification: Biological and translational research implications

(2014) K. Anthony et al.   NEUROLOGY

Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy

(2014) Andrew P. Lieberman et al.   Cell Reports

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

(2014) Qi-long Lu et al.   Molecular Therapy-Nucleic Acids

A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

(2013) Haiyan Zhou et al.   HUMAN GENE THERAPY

Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy

(2013) Paul N. Porensky et al.   HUMAN GENE THERAPY

Meta-analysis of data from animal studies: A practical guide

(2013) H.M. Vesterinen et al.   JOURNAL OF NEUROSCIENCE METHODS

Letter to the Editor

(2013) Rob B M de Vries et al.   LABORATORY ANIMALS

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Splicing therapy for neuromuscular disease

(2013) Andrew G.L. Douglas et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

(2013) C. Lagier-Tourenne et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice

(2013) Silvana M.G. Jirka et al.   Nucleic Acid Therapeutics

Threats to Validity in the Design and Conduct of Preclinical Efficacy Studies: A Systematic Review of Guidelines for In Vivo Animal Experiments

(2013) Valerie C. Henderson et al.   PLOS MEDICINE

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency

(2013) HaiFang Yin et al.   Molecular Therapy-Nucleic Acids

Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic mdx Mice by Peptide-Conjugated Morpholino

(2012) Bo Wu et al.   AMERICAN JOURNAL OF PATHOLOGY

Cell-penetrating peptides for the delivery of nucleic acids

(2012) Taavi Lehto et al.   Expert Opinion on Drug Delivery

Antisense Oligonucleotides: Rising Stars in Eliminating RNA Toxicity in Myotonic Dystrophy

(2012) Zhihua Gao et al.   HUMAN GENE THERAPY

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

(2012) Adeline Vulin et al.   MOLECULAR THERAPY

A call for transparent reporting to optimize the predictive value of preclinical research

(2012) Story C. Landis et al.   NATURE

Raise standards for preclinical cancer research

(2012) C. Glenn Begley et al.   NATURE

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle

(2011) Eric P. Hoffman et al.   AMERICAN JOURNAL OF PATHOLOGY

A step-by-step guide to systematically identify all relevant animal studies

(2011) Marlies Leenaars et al.   LABORATORY ANIMALS

An octaguanidine-morpholino oligo conjugate improves muscle function of mdx mice

(2011) Jeffrey J. Widrick et al.   MUSCLE & NERVE

Antisense-Mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain

(2011) Ioannis Zalachoras et al.   Frontiers in Molecular Neuroscience

Enhancing search efficiency by means of a search filter for finding all studies on animal experimentation in PubMed

(2010) Carlijn R Hooijmans et al.   LABORATORY ANIMALS

Improving Bioscience Research Reporting: The ARRIVE Guidelines for Reporting Animal Research

(2010) Carol Kilkenny et al.   PLOS BIOLOGY

Mammalian animal models for Duchenne muscular dystrophy

(2009) Raffaella Willmann et al.   NEUROMUSCULAR DISORDERS

AAV vectors for RNA-based modulation of gene expression

(2008) O Danos   GENE THERAPY

Cell penetrating peptide conjugates of steric block oligonucleotides

(2007) Bernard Lebleu et al.   ADVANCED DRUG DELIVERY REVIEWS