New antisense oligonucleotide therapies reach first base in ALS

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

(2022) Vladislav A. Korobeynikov et al.   NATURE MEDICINE

Double setback for ASO trials in Huntington disease

(2021) Katie Kingwell   NATURE REVIEWS DRUG DISCOVERY

An oral antisense oligonucleotide for PCSK9 inhibition

(2021) Peter Gennemark et al.   Science Translational Medicine

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

(2021) Tetsuya Nagata et al.   NATURE BIOTECHNOLOGY

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

(2021) Hélène Tran et al.   NATURE MEDICINE

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

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

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

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

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

(2011) Mariely DeJesus-Hernandez et al.   NEURON

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

(2011) Alan E. Renton et al.   NEURON