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

A large animal model of spinal muscular atrophy and correction of phenotype.

Annals of neurology - 1 Mar 2015

Duque Sandra I, Arnold W David, Odermatt Philipp, Li Xiaohui, Porensky Paul N, Schmelzer Leah, Meyer Kathrin, Kolb Stephen J, Schümperli Daniel, Kaspar Brian K, Burghes Arthur H M

Abstract excerpt

OBJECTIVES: Spinal muscular atrophy (SMA) is caused by reduced levels of survival motor neuron (SMN) protein, which results in motoneuron loss. Therapeutic strategies to increase SMN levels including drug compounds, antisense oligonucleotides, and scAAV9 gene therapy have proved effective in mice. We wished to determine whether reduction of SMN in postnatal motoneurons resulted in SMA in a large animal model,...

Read the complete abstract on PubMed

Topics

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

Share to Dependovirus
Share to Electromyography

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Genetic Therapy

Share to Genetic Vectors

Share to Motor Neurons

Share to Muscular Atrophy, Spinal

Share to RNA, Small Interfering

Share to SMN Complex Proteins

Source

Open in PubMed Open full text

Identifiers

PMID: 25516063
DOI: 10.1002/ana.24332
PMCID: PMC4453930

Genes

SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

A large animal model of spinal muscular atrophy and correction of phenotype.PMID 25516063Read ↗

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophySemantic proximity Read ↗

Therapeutic strategies for spinal muscular atrophy: SMN and beyondSemantic proximity Read ↗

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.Semantic proximity Read ↗

Decreasing Disease Severity in Symptomatic, <i>Smn</i> <sup>−/−</sup> ; <i>SMN2</i> <sup>+/+</sup> , Spinal Muscular Atrophy Mice Following scAAV9-SMN DeliverySemantic proximity Read ↗

Motor Neuron Rescue in Spinal Muscular Atrophy Mice Demonstrates That Sensory-Motor Defects Are a Consequence, Not a Cause, of Motor Neuron DysfunctionSemantic proximity Read ↗

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophySemantic proximity Read ↗

Spinal muscular atrophy: from tissue specificity to therapeutic strategiesSemantic proximity Read ↗

SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMASemantic proximity Read ↗

Disease mechanisms and therapeutic approaches in spinal muscular atrophy.Semantic proximity Read ↗

Moving towards treatments for spinal muscular atrophy: hopes and limitsSemantic proximity Read ↗

Improvement of neuromuscular synaptic phenotypes without enhanced survival and motor function in severe spinal muscular atrophy mice selectively rescued in motor neurons.Semantic proximity Read ↗

Defining the therapeutic window in a severe animal model of spinal muscular atrophy.Semantic proximity Read ↗

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