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

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.

Human molecular genetics - 15 Feb 2011

Dominguez Elisa, Marais Thibaut, Chatauret Nicolas, Benkhelifa-Ziyyat Sofia, Duque Sandra, Ravassard Philippe, Carcenac Romain, Astord Stéphanie, Pereira de Moura Aurélie, Voit Thomas, Barkats Martine

Abstract excerpt

Spinal muscular atrophy (SMA) is the most common genetic disease leading to infant mortality. This neuromuscular disorder is caused by the loss or mutation of the telomeric copy of the 'survival of motor neuron' (Smn) gene, termed SMN1. Loss of SMN1 leads to reduced SMN protein levels, inducing d...

Read the complete abstract on PubMed

Topics

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

Share to Dependovirus
Share to Gene Transfer Techniques
Share to Genetic Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Motor Neurons

Share to Muscular Atrophy, Spinal

Share to Spinal Cord

Share to Survival Analysis

Share to Survival of Motor Neuron 1 Protein

Source

Identifiers

PMID: 21118896
DOI: 10.1093/hmg/ddq514

Genes

SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-21118896-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.PMID 21118896Read ↗

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 ↗

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

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA MiceSemantic proximity Read ↗

Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and ChallengesSemantic proximity Read ↗

Update on gene and stem cell therapy approaches for spinal muscular atrophy.Semantic proximity Read ↗

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

Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in MiceSemantic proximity Read ↗

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.Semantic proximity Read ↗

[SMA: from gene discovery to gene therapy].Semantic proximity Read ↗

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotorSemantic proximity Read ↗

An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular AtrophySemantic proximity Read ↗

Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophySemantic proximity Read ↗

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