Nucleic Acid Therapeutics for Neurological Diseases

Oligonucleotide Therapeutics as a New Class of Drugs for Malignant Brain Tumors: Targeting mRNAs, Regulatory RNAs, Mutations, Combinations, and Beyond

(2019) Anna M. Krichevsky et al.   Neurotherapeutics

Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias

(2019) Ronald A.M. Buijsen et al.   Neurotherapeutics

Nucleic Acid Therapies for Ischemic Stroke

(2019) Nils Henninger et al.   Neurotherapeutics

Nucleic Acid–Based Therapeutics for Parkinson’s Disease

(2019) Masayuki Nakamori et al.   Neurotherapeutics

Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases

(2019) Rula Zain et al.   Neurotherapeutics

Nucleic Acid-Based Therapeutics Relevant to Neuroimmune Conditions

(2019) Ariele L. Greenfield et al.   Neurotherapeutics

Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction

(2019) Michelle L. Hastings et al.   Neurotherapeutics

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

A tailormade drug developed in record time may save girl from fatal brain disease

(2018) Jocelyn Kaiser   SCIENCE

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

(2017) Annemieke Aartsma-Rus   Nucleic Acid Therapeutics

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

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

(2010) Frederick J Raal et al.   LANCET