Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome

4 Aug 2021

Abstract excerpt

Intronic splicing silencer N1 (ISS-N1) located within Survival Motor Neuron 2 (SMN2) intron 7 is the target of a therapeutic antisense oligonucleotide (ASO), nusinersen (Spinraza), which is currently being used for the treatment of spinal muscular atrophy (SMA), a leading genetic disease associated with infant mortality. The discovery of ISS-N1 as a promising therapeutic target was enabled in part by Anti-N1, a...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Neurogenetic and Muscular Disorders Research
Share to RNA modifications and cancer
Share to RNA Research and Splicing

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 34445083
DOI: 10.3390/ijms22168378

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the TranscriptomePMID 34445083Read ↗

Antisense oligonucleotide mediated therapy of spinal muscular atrophySemantic proximity Read ↗

RNA in spinal muscular atrophy: therapeutic implications of targeting.Semantic proximity Read ↗

Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy.Semantic proximity Read ↗

Nusinersen in the Treatment of Spinal Muscular Atrophy.Semantic proximity Read ↗

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscapeSemantic proximity Read ↗

A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular AtrophySemantic proximity Read ↗

Towards a combined therapy for spinal muscular atrophy based on opposing effects of an antisense oligonucleotide on chromatin and splicingSemantic proximity Read ↗

Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy.Semantic proximity Read ↗

Preliminary insights into RNA in CSF of pediatric SMA patients after 6 months of nusinersen.Semantic proximity Read ↗

Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular AtrophySemantic proximity Read ↗

Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.Semantic proximity Read ↗

Antisense Oligonucleotides for the Treatment of Spinal Muscular AtrophySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.