ReviewCellular and molecular life sciences : CMLS2012
Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis.
Review in Cellular and molecular life sciences : CMLS, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed.
- Synaptotagmin 13 is neuroprotective across motor neuron diseases.Acta neuropathologica · 2020Article
- Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Frontiers in molecular neuroscience · 2017Review
- Neuroplasticity and Repair in Rodent Neurotoxic Models of Spinal Motoneuron Disease.Neural plasticity · 2016Review
- Stem cell transplantation for amyotrophic lateral sclerosis: therapeutic potential and perspectives on clinical translation.Cellular and molecular life sciences : CMLS · 2014Review
- RNA-binding proteins associated molecular mechanisms of motor neuron degeneration pathogenesis.Molecular biotechnology · 2014Review
- Generating spinal motor neuron diversity: a long quest for neuronal identity.Cellular and molecular life sciences : CMLS · 2014Review
- A missense mutation in AGTPBP1 was identified in sheep with a lower motor neuron disease.Heredity · 2012Article
- AAV2 mediated retrograde transduction of corticospinal motor neurons reveals initial and selective apical dendrite degeneration in ALS.Neurobiology of disease · 2012Article
- The advent of AAV9 expands applications for brain and spinal cord gene delivery.Expert opinion on biological therapy · 2012Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease of motor neurons that causes progressive muscle weakness, paralysis, and premature death. No effective therapy is available. Research in the motor neuron field continues to grow, and recent breakthroughs have demonstrated the possibility of completely achieving rescue in animal models of spinal muscular atrophy, a genetic motor neuron disease. With adeno-associated virus (AAV) vectors, gene transfer can be achieved with systemic non-invasive injection and minimal toxicity. In the context of this success, we review gene therapy approaches for ALS, considering what has been done and the possible future directions for effective application of the latest generation of vectors for clinical translation. We focus on recent developments in the areas of RNA/antisense-mediated silencing of specific ALS causative genes like superoxide dismutase-1 and other molecular pathogenetic targets, as well as the administration of neuroprotective factors with viral vectors. We argue that gene therapy offers new opportunities to open the path for clinical progress in treating ALS.
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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.