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

Update on gene and stem cell therapy approaches for spinal muscular atrophy.

Expert opinion on biological therapy - 1 Nov 2012

Donnelly Eleanor M, Boulis Nicholas M

Abstract excerpt

INTRODUCTION: Spinal muscular atrophy (SMA) is the leading genetic cause of pediatric death to which at present there is no effective therapeutic. The genetic defect is well characterized as a mutation in exon 7 of the survival of motor neuron (SMN) gene. The current gene therapy approach focuses on two main methodologies, the replacement of SMN1 or augmentation of SMN2 readthrough. The most promising of the...

Read the complete abstract on PubMed

Topics

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

Share to Clinical Trials as Topic
Share to Genetic Therapy
Share to Genetic Vectors

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Atrophy, Spinal

Share to Mutation

Share to Stem Cell Transplantation

Share to Survival of Motor Neuron 1 Protein

Share to Survival of Motor Neuron 2 Protein

Source

Identifiers

PMID: 22849423
DOI: 10.1517/14712598.2012.711306

Genes

SMN1
SMN2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Update on gene and stem cell therapy approaches for spinal muscular atrophy.PMID 22849423Read ↗

Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA).Semantic proximity Read ↗

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent LandscapeSemantic proximity Read ↗

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

New Directions for SMA TherapySemantic proximity Read ↗

In utero therapy for spinal muscular atrophy: closer to clinical translationSemantic proximity Read ↗

Advances in Therapeutic Development for Spinal Muscular AtrophySemantic proximity Read ↗

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

New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?Semantic proximity Read ↗

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

Gene therapy in spinal muscular atrophy.Semantic proximity Read ↗

Current and emerging targeted therapies for spinal muscular atrophy.Semantic proximity Read ↗

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

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