ReviewJournal of cellular and molecular medicine2014
Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA).
Review in Journal of cellular and molecular medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed.
- Therapeutic potential of stem cells in pediatric neurology: Insights from clinical trials.Neuroprotection (Chichester, England) · 2025Review
- Patient and Caregiver Treatment Preferences in Type 2 and Non-ambulatory Type 3 Spinal Muscular Atrophy: A Discrete Choice Experiment Survey in Five European Countries.PharmacoEconomics · 2022Article
- A narrative review of in utero gene therapy: advances, challenges, and future considerations.Translational pediatrics · 2021Review
- Validation of a Fast, Robust, Inexpensive, Two-Tiered Neonatal Screening Test algorithm on Dried Blood Spots for Spinal Muscular Atrophy.International journal of neonatal screening · 2019Article
- A Semi-Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.CPT: pharmacometrics & systems pharmacology · 2018Article
- Advances in spinal muscular atrophy therapeutics.Therapeutic advances in neurological disorders · 2018Review
- Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Neuron · 2017Review
- Is spinal muscular atrophy a disease of the motor neurons only: pathogenesis and therapeutic implications?Cellular and molecular life sciences : CMLS · 2016Review
- Review of Spinal Muscular Atrophy (SMA) for Prenatal and Pediatric Genetic Counselors.Journal of genetic counseling · 2016Article
- Neuroplasticity and Repair in Rodent Neurotoxic Models of Spinal Motoneuron Disease.Neural plasticity · 2016Review
- Neuroprotective Effect of Non-viral Gene Therapy Treatment Based on Tetanus Toxin C-fragment in a Severe Mouse Model of Spinal Muscular Atrophy.Frontiers in molecular neuroscience · 2016Article
- Clinical and molecular features and therapeutic perspectives of spinal muscular atrophy with respiratory distress type 1.Journal of cellular and molecular medicine · 2015Review
- Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.Scientific reports · 2015Article
- Spinal muscular atrophy: therapeutic strategies.Current treatment options in neurology · 2014Article
- Human-induced pluripotent stem cells: potential for neurodegenerative diseases.Human molecular genetics · 2014Review
- Yeast Augmented Network Analysis (YANA): a new systems approach to identify therapeutic targets for human genetic diseases.F1000Research · 2014Article
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease. It is the first genetic cause of infant mortality. It is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the reduction of SMN protein. The most striking component is the loss of alpha motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment other than supportive care, although the past decade has seen a striking advancement in understanding of both SMA genetics and molecular mechanisms. A variety of disease modifying interventions are rapidly bridging the translational gap from the laboratory to clinical trials. In this review, we would like to outline the most interesting therapeutic strategies that are currently developing, which are represented by molecular, gene and stem cell-mediated approaches for the treatment of SMA.
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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.