Back to search

Article

Type I PRMT inhibitor MS023 promotes <i>SMN2</i> exon 7 inclusion and synergizes with nusinersen to rescue the phenotype of SMA mice

2022-10-18

Abstract excerpt

Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality. The advent of approved treatments for this devastating condition has significantly changed SMA patients’ life expectancy and quality of life. Nevertheless, these are not without limitations, and research efforts are underway to develop new approaches to be used alone and in combination, to ensure improved and long-lasting benefits for...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
132e6b0d-8a5a-5d40-9691-47151b059266
DOI
10.1101/2022.10.18.512489
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
Type I PRMT inhibitor MS023 promotes <i>SMN2</i> exon 7 inclusion and synergizes with nusinersen to rescue the phenotype of SMA miceDOI 10.1101/2022.10.18.512489
Towards a combined therapy for spinal muscular atrophy based on opposing effects of an antisense oligonucleotide on chromatin and splicingSemantic proximity
<i>SMN2</i> splicing modifiers improve motor function and longevity in mice with spinal muscular atrophySemantic proximity
Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscapeSemantic proximity
Pathogenesis and therapeutic targets in spinal muscular atrophy (SMA).Semantic proximity
RNA in spinal muscular atrophy: therapeutic implications of targeting.Semantic proximity
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular AtrophySemantic proximity
Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)Semantic proximity
Mechanistic studies of a small-molecule modulator of SMN2 splicingSemantic proximity
SMN-Inducing Compounds For The Treatment Of Spinal Muscular AtrophySemantic proximity
New Directions for SMA TherapySemantic proximity
Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy TherapySemantic proximity
Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophySemantic proximity
Select a neighboring publication to make it the new centre.