Sage Journals: Discover world-class research

Retricted access

Review article

First published 2013-05

Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy

Paul N. Porensky, Arthur H.M. Burghes

Human Gene Therapy

Open access

Review article

First published 2026-3

A historical perspective on the development of antisense oligonucleotide treatments for Duchenne muscular dystrophy and spinal muscular atrophy

Annemieke Aartsma-Rus, Shin'ichi Takeda

Journal of Neuromuscular Diseases

Open access

Review article

First published 2019-2

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

Michael J. Gait, Andrey A. Arzumanov, Graham Mcclorey [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2007-8

Immune Modulatory Oligonucleotides in the Prevention and Treatment of OVA-Induced Eustachian Tube Dysfunction in Rats

Deidra A. Blanks, Charles S. Ebert, Rose P. Eapen [...]

Otolaryngology–Head and Neck Surgery

Open access

Research article

First published 2022-04

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic,Regulatory,and Ethical Perspectives

Matthis Synofzik, Willeke M.C van Roon-Mom, Georg Marckmann [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2016-08

Effects of Repeated Complement Activation Associated with Chronic Treatment of Cynomolgus Monkeys with 2′- O -Methoxyethyl Modified Antisense Oligonucleotide

Lijiang Shen, Jeffrey A. Engelhardt, Gene Hung [...]

Nucleic Acid Therapeutics

Open access

Review article

First published 2024

Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

Annemieke Aartsma-Rus, Rob W.J. Collin, Ype Elgersma [...]

Therapeutic Advances in Rare Disease

Retricted access

Research article

First published 1998-04

Non-Antisense Oligonucleotide Approaches for Experimental Treatment of Glioblastoma

Len M. Neckers, Meera Kanekal, Yvette Connell

Nucleic Acid Therapeutics

Free access

Research article

First published 2004-8

Virological Effects of Isis 14803,An Antisense Oligonucleotide Inhibitor of Hepatitis C Virus (HCV) Internal Ribosome Entry Site (IRES),on HCV Ires in Chronic Hepatitis C Patients and Examination of the Potential Role of Primary and Secondary HCV Resistance in the Outcome of Treatment

Muriel Soler, John G McHutchison, T Jesse Kwoh [...]

Antiviral Therapy

Open access

Research article

First published 2025-08

Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection

Jurriën Prins, Janneke Kouwenberg, Anouk Spruit [...]

Nucleic Acid Therapeutics

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