Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS

Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease

(2018) Tracy L. Hagemann et al.   ANNALS OF NEUROLOGY

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

(2018) Alex McCampbell et al.   JOURNAL OF CLINICAL INVESTIGATION

Angubindin-1 opens the blood–brain barrier in vivo for delivery of antisense oligonucleotide to the central nervous system

(2018) Satoshi Zeniya et al.   JOURNAL OF CONTROLLED RELEASE

Antisense-mediated reduction of EphA4 in the adult CNS does not improve the function of mice with amyotrophic lateral sclerosis

(2018) Karen K. Ling et al.   NEUROBIOLOGY OF DISEASE

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

Current Strategies for Brain Drug Delivery

(2018) Xiaowei Dong   Theranostics

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

(2018) Annabelle Biscans et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases

(2017) Kathleen M. Schoch et al.   NEURON

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

(2017) Fazel Shabanpoor et al.   Nucleic Acid Therapeutics

Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice

(2017) Dominic Jauvin et al.   Molecular Therapy-Nucleic Acids

Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model

(2017) Karima Relizani et al.   Molecular Therapy-Nucleic Acids

Oligonucleotide‐induced alternative splicing of serotonin 2C receptor reduces food intake

(2016) Zhaiyi Zhang et al.   EMBO Molecular Medicine

Evaluation of the effects of chemically different linkers on hepatic accumulations, cell tropism and gene silencing ability of cholesterol-conjugated antisense oligonucleotides

(2016) Shunsuke Wada et al.   JOURNAL OF CONTROLLED RELEASE

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

(2016) Suzan M. Hammond et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Oligonucleotide Therapies: The Past and the Present

(2015) Karin E. Lundin et al.   HUMAN GENE THERAPY

DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing

(2015) Kazutaka Nishina et al.   Nature Communications

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

(2015) Tomoko Nishina et al.   Molecular Therapy-Nucleic Acids

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

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Consensus on cerebral involvement in myotonic dystrophy

(2014) E. Bugiardini et al.   NEUROMUSCULAR DISORDERS

Electroporation and Microinjection Successfully Deliver Single-Stranded and Duplex DNA into Live Cells as Detected by FRET Measurements

(2014) Rosemary A. Bamford et al.   PLoS One

Natural course of scoliosis in proximal spinal muscular atrophy type II and IIIa: descriptive clinical study with retrospective data collection of 126 patients

(2013) Albert Fujak et al.   BMC MUSCULOSKELETAL DISORDERS

Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals

(2013) Gene Hung et al.   Nucleic Acid Therapeutics

Childhood spinal muscular atrophy: controversies and challenges

(2012) Eugenio Mercuri et al.   LANCET NEUROLOGY

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

Specific Detection of Pathological Three-repeat Tau after Pretreatment with Potassium Permanganate and Oxalic Acid in PSP/CBD Brains

(2010) Toshiki Uchihara et al.   BRAIN PATHOLOGY

The MIQE Guidelines: Minimum Information for Publication of Quantitative Real-Time PCR Experiments

(2009) S. A. Bustin et al.   CLINICAL CHEMISTRY

Antisense oligonucleotide pharmacokinetics and metabolism

(2009) Richard S Geary   Expert Opinion on Drug Metabolism & Toxicology

DMPK protein isoforms are differentially expressed in myogenic and neural cell lineages

(2009) Ralph J. A. Oude Ophuis et al.   MUSCLE & NERVE

Spinal muscular atrophy

(2008) Mitchell R Lunn et al.   LANCET