ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Specific retrograde transduction of spinal motor neurons using lentiviral vectors targeted to presynaptic NMJ receptors.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 22 citations in OpenAlex.
- Properties and Application of Pseudotyped Lentiviral Vectors.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Intramuscular Delivery of Gene Therapy for Targeting the Nervous System.Frontiers in molecular neuroscience · 2020Article
- Motor neuron degeneration following glycine-mediated excitotoxicity induces spastic paralysis after spinal cord ischemia/reperfusion injury in rabbit.American journal of translational research · 2017Article
- A novel approach for targeted delivery to motoneurons using cholera toxin-B modified protocells.Journal of neuroscience methods · 2016Article
- PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer's disease model.Proceedings of the National Academy of Sciences of the United States of America · 2016Article
- Article
- Targeting Motor End Plates for Delivery of Adenoviruses: An Approach to Maximize Uptake and Transduction of Spinal Cord Motor Neurons.Scientific reports · 2016Article
- A new glucocerebrosidase-deficient neuronal cell model provides a tool to probe pathophysiology and therapeutics for Gaucher disease.Disease models & mechanisms · 2016Article
- Microfluidic Organ/Body-on-a-Chip Devices at the Convergence of Biology and Microengineering.Sensors (Basel, Switzerland) · 2015Review
- A compartmentalized microfluidic neuromuscular co-culture system reveals spatial aspects of GDNF functions.Journal of cell science · 2015Article
- Gene delivery strategies to promote spinal cord repair.Biomarker insights · 2015Review
- Rabies virus envelope glycoprotein targets lentiviral vectors to the axonal retrograde pathway in motor neurons.The Journal of biological chemistry · 2014Article
- Non-viral gene therapy that targets motor neurons in vivo.Frontiers in molecular neuroscience · 2014Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
To understand how receptors are involved in neuronal trafficking and to be able to utilize them for specific targeting via the peripheral route would be of great benefit. Here, we describe the generation of novel lentiviral vectors with tropism to motor neurons that were made by coexpressing onto the lentiviral surface a fusogenic glycoprotein (mutated sindbis G) and an antibody against a cell-surface receptor (Thy1.1, p75(NTR), or coxsackievirus and adenovirus receptor) on the presynaptic terminal of the neuromuscular junction. These vectors exhibit binding specificity and efficient transduction of receptor positive cell lines and primary motor neurons in vitro. Targeting of each of these receptors conferred to these vectors the capability of being transported retrogradely from the axonal tip, leading to transduction of motor neurons in vitro in compartmented microfluidic cultures. In vivo delivery of coxsackievirus and adenovirus receptor-targeted vectors in leg muscles of mice resulted in predicted patterns of motor neuron labeling in lumbar spinal cord. This opens up the clinical potential of these vectors for minimally invasive administration of central nervous system-targeted therapeutics in motor neuron diseases.
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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.