ArticleThe Journal of neuroscience : the official journal of the Society for Neuroscience2009
Motoneuron transplantation rescues the phenotype of SMARD1 (spinal muscular atrophy with respiratory distress type 1).
Article in The Journal of neuroscience : the official journal of the Society for Neuroscience, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 39 citations in OpenAlex.
- AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model.Journal of biomedical science · 2026Article
- Disease Mechanisms and Therapeutic Approaches in SMARD1-Insights from Animal Models and Cell Models.Biomedicines · 2024Review
- Current understanding of and emerging treatment options for spinal muscular atrophy with respiratory distress type 1 (SMARD1).Cellular and molecular life sciences : CMLS · 2020Review
- Spinal muscular atrophy with respiratory distress type 1: Clinical phenotypes, molecular pathogenesis and therapeutic insights.Journal of cellular and molecular medicine · 2020Review
- Advances, Challenges, and Perspectives in Translational Stem Cell Therapy for Amyotrophic Lateral Sclerosis.Molecular neurobiology · 2019Review
- Neuromuscular Junctions as Key Contributors and Therapeutic Targets in Spinal Muscular Atrophy.Frontiers in neuroanatomy · 2016Review
- Clinical and molecular features and therapeutic perspectives of spinal muscular atrophy with respiratory distress type 1.Journal of cellular and molecular medicine · 2015Review
- Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.Science advances · 2015Article
- Truncating and missense mutations in IGHMBP2 cause Charcot-Marie Tooth disease type 2.American journal of human genetics · 2014Article
- iPSC-Derived neural stem cells act via kinase inhibition to exert neuroprotective effects in spinal muscular atrophy with respiratory distress type 1.Stem cell reports · 2014Article
- Generating spinal motor neuron diversity: a long quest for neuronal identity.Cellular and molecular life sciences : CMLS · 2014Review
- Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy.Science translational medicine · 2012Article
- Human pluripotent stem cells: applications and challenges in neurological diseases.Frontiers in physiology · 2012Article
- Spinal muscular atrophy.Orphanet journal of rare diseases · 2011Review
- Stem cell transplantation for motor neuron disease: current approaches and future perspectives.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2011Review
- Human motor neuron progenitor transplantation leads to endogenous neuronal sparing in 3 models of motor neuron loss.Stem cells international · 2011Article
- Human motor neuron generation from embryonic stem cells and induced pluripotent stem cells.Cellular and molecular life sciences : CMLS · 2010Review
- Amyotrophic lateral sclerosis: applications of stem cells - an update.Stem cells and cloning : advances and applications · 2010Review
- Human stem cells as a model of motoneuron development and diseases.Annals of the New York Academy of Sciences · 2010Article
- Spinal muscular atrophy: mechanisms and therapeutic strategies.Human molecular genetics · 2010Review
Corrections and comments
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Authors and funding
12 authors at 4 institutions in 1 country.
Funding
Abstract
Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a fatal form of infantile motoneuron disease. There is currently no effective treatment, although motor neuron replacement is a possible therapeutic strategy. We transplanted purified motor neurons into the spinal cord of nmd mice, an animal model of SMARD1. We also administered pharmacological treatment targeting the induction of axonal growth toward skeletal muscle target. At the end stage of the disease, donor-derived motor neurons were detected in the nmd anterior horns, extended axons into the ventral roots, and formed new neuromuscular junctions. These data correlated with improved neuromuscular function and increased life spans. The neuroprotective effect was associated with a reduction in proinflammatory molecules in treated spinal cords. This is the first report that functional restoration of motor units with transplanted motoneurons is feasible in an animal model of a human motoneuron disease, opening up new possibilities for therapeutic intervention.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.