The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Convective forces increase rostral delivery of intrathecal radiotracers and antisense oligonucleotides in the cynomolgus monkey nervous system

(2020) Jenna M. Sullivan et al.   Journal of Translational Medicine

Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease

(2020) Matthew S. Elitt et al.   NATURE

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

(2020) Timothy Miller et al.   NEW ENGLAND JOURNAL OF MEDICINE

Selective tissue targeting of synthetic nucleic acid drugs

(2019) Punit P. Seth et al.   JOURNAL OF CLINICAL INVESTIGATION

RNA-Targeted Therapeutics

(2019) Stanley T. Crooke et al.   Cell Metabolism

Targeting Huntingtin Expression in Patients with Huntington’s Disease

(2019) Sarah J. Tabrizi et al.   NEW ENGLAND JOURNAL OF MEDICINE

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

(2019) Julia F. Alterman et al.   NATURE BIOTECHNOLOGY

Targeting microglia with lentivirus and AAV: Recent advances and remaining challenges

(2019) Margaret E. Maes et al.   NEUROSCIENCE LETTERS

Alzheimer Disease: An Update on Pathobiology and Treatment Strategies

(2019) Justin M. Long et al.   CELL

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

(2019) Daniel M. Ramos et al.   JOURNAL OF CLINICAL INVESTIGATION

Oligonucleotide therapy mitigates disease in Spinocerebellar Ataxia Type 3 mice

(2018) Hayley S. McLoughlin et al.   ANNALS OF NEUROLOGY

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

Spinocerebellar ataxias: prospects and challenges for therapy development

(2018) Tetsuo Ashizawa et al.   Nature Reviews Neurology

Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7

(2018) Chenchen Niu et al.   Science Translational Medicine

Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease

(2018) Amber L. Southwell et al.   Science Translational Medicine

Targeted delivery of antisense oligonucleotides to pancreatic β-cells

(2018) C. Ämmälä et al.   Science Advances

Development of a simple, rapid, and robust intrathecal catheterization method in the rat

(2017) Curt Mazur et al.   JOURNAL OF NEUROSCIENCE METHODS

Pharmacokinetics of an intracerebroventricularly administered antibody in rats

(2017) Yuki Noguchi et al.   mAbs

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

(2017) Lindsay A. Becker et al.   NATURE

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy

(2017) Sarah L. DeVos et al.   Science Translational Medicine

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

(2017) Hien Tran Zhao et al.   Molecular Therapy-Nucleic Acids

Comprehensive Structure–Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes

(2016) Thazha P. Prakash et al.   JOURNAL OF MEDICINAL CHEMISTRY

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

(2016) Richard S Finkel et al.   LANCET

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

(2016) Jie Jiang et al.   NEURON

Contribution of the oligodendrocyte lineage to CNS repair and neurodegenerative pathologies

(2016) Reshmi Tognatta et al.   NEUROPHARMACOLOGY

Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides

(2015) Yehezkel Sztainberg et al.   NATURE

Molecular determinants of selective dopaminergic vulnerability in Parkinson’s disease: an update

(2014) Lars Brichta et al.   Frontiers in Neuroanatomy

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

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

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

(2012) Holly B. Kordasiewicz et al.   NEURON

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT