ArticleGene therapy2013
Efficient gene expression from integration-deficient lentiviral vectors in the spinal cord.
Article in Gene therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed.
- In utero therapy for spinal muscular atrophy: closer to clinical translation.Brain : a journal of neurology · 2025Review
- Review
- Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo.Gene therapy · 2023Article
- Review
- Article
- Improved Functionality of Integration-Deficient Lentiviral Vectors (IDLVs) by the Inclusion of ISPharmaceutics · 2021Article
- Review
- Safe and neuroprotective vectors for long-term traumatic brain injury gene therapy.Gene therapy · 2020Article
- On the development of optical peripheral nerve interfaces.Neural regeneration research · 2019Review
- High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors.Journal of controlled release : official journal of the Controlled Release Society · 2018Article
- Effective gene expression in the rat dorsal root ganglia with a non-viral vector delivered via spinal nerve injection.Scientific reports · 2016Article
- Developing therapies for spinal muscular atrophy.Annals of the New York Academy of Sciences · 2016Review
- Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors.Molecular therapy. Methods & clinical development · 2015Article
- Expression of suppressor of cytokine signaling-3 (SOCS3) and its role in neuronal death after complete spinal cord injury.Experimental neurology · 2014Article
- Cell-based therapies for Huntington's disease.Drug discovery today · 2014Review
- Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease.Human gene therapy · 2014Article
- Targeted gene therapy and cell reprogramming in Fanconi anemia.EMBO molecular medicine · 2014Article
- Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors.Human gene therapy · 2014Article
- Lentiviral vector-mediated RNA silencing in the central nervous system.Human gene therapy methods · 2014Review
- Design and Potential of Non-Integrating Lentiviral Vectors.Biomedicines · 2014Review
Corrections and comments
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Authors and funding
13 authors.
Funding
Abstract
Gene transfer to spinal cord cells may be crucial for therapy in spinal muscular atrophy, amyotrophic lateral sclerosis and spinal cord injury. Lentiviral vectors are efficient for transduction of a variety of cells, but like all integrating vectors they pose a risk of insertional mutagenesis. Integration-deficient lentiviral vectors (IDLVs) remain episomal but retain the transduction efficiency of standard integrating lentiviral vectors, particularly when the episomes are not diluted out through repeated cell division. We have now applied IDLVs for transduction of spinal cord in vitro, in explants and in vivo. Our results demonstrate similar efficiency of eGFP expression from integrating lentiviral vectors and IDLVs in most cell types analyzed, including motor neurons, interneurons, dorsal root ganglia (DRG) neurons and astroglia. IDLV-mediated expression of pro-glial-cell-derived neurotrophic factor (Gdnf) rescues motor neuron cultures from death caused by removal of exogenous trophic support. IDLVs also mediate efficient RNA interference in DRG neuron cultures. After intraparenchymal injection in the rat and mouse cervical and lumbar regions in vivo, transduction is mainly neuronal, with both motor neurons and interneurons being efficiently targeted. These results suggest that IDLVs could be efficient and safer tools for spinal cord transduction in future therapeutic strategies.
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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.