ArticleHuman gene therapy2011
Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.
Article in Human gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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.
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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.
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Who cites it
19 citing papers in PubMed, 43 citations in OpenAlex.
- Optogenetic Approach for In Vivo Manipulation of Neural Circuits Using Lentiviral Vectors.Methods in molecular biology (Clifton, N.J.) · 2026Article
- A Risk Factor for Attention Deficit Hyperactivity Disorder Induces Marked Long-Term Anatomical Changes at GABAergic-Dopaminergic Synapses in the Rat Ventral Tegmental Area.International journal of molecular sciences · 2024Article
- Therapeutic CRISPR epigenome editing of inflammatory receptors in the intervertebral disc.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Efficacy of dual intracerebroventricular and intravitrealFrontiers in pharmacology · 2023Article
- Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease.Frontiers in genetics · 2023Article
- Selecting the Best Animal Model of Parkinson's Disease for Your Research Purpose: Insight fromCurrent neuropharmacology · 2023Article
- The Sheep as a Large Animal Model for the Investigation and Treatment of Human Disorders.Biology · 2022Review
- Article
- A lysosomal enigma CLN5 and its significance in understanding neuronal ceroid lipofuscinosis.Cellular and molecular life sciences : CMLS · 2021Review
- Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain.Molecular therapy. Methods & clinical development · 2019Article
- Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Efficient Transduction and Expansion of Ovine Macrophages for Gene Therapy Implementations.Veterinary sciences · 2018Article
- Gene therapy approaches in the non-human primate model of Parkinson's disease.Journal of neural transmission (Vienna, Austria : 1996) · 2018Review
- RNA-Sequencing Analysis Reveals a Regulatory Role for Transcription FactorFrontiers in molecular neuroscience · 2017Article
- Viral vector-based tools advance knowledge of basal ganglia anatomy and physiology.Journal of neurophysiology · 2016Review
- Chimeric rabies SADB19-VSVg-pseudotyped lentiviral vectors mediate long-range retrograde transduction from the mouse spinal cord.Gene therapy · 2015Article
- Patterned, but not tonic, optogenetic stimulation in motor thalamus improves reaching in acute drug-induced Parkinsonian rats.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2015Article
- Article
- Lentiviral vectors as tools to understand central nervous system biology in mammalian model organisms.Frontiers in molecular neuroscience · 2015Review
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 at 2 institutions in 1 country.
Funding
Abstract
The neuronal ceroid lipofuscinoses (NCLs; Batten disease) are inherited neurodegenerative lysosomal storage diseases with common clinical features of blindness and seizures culminating in premature death. Gene-therapy strategies for these diseases depend on whether the missing activity is a secreted lysosomal protein taken up by neighboring cells, or an intramembrane protein that requires careful targeting. Therapies are best developed in animal models with large complex human-like brains. Lentiviral-mediated gene delivery to neural cell cultures from normal sheep and sheep affected with an NCL resulted in green fluorescent protein (GFP) expression in neurons and neuroblasts, more efficiently than in astrocytes. Similar transgene expression was obtained from two constitutive promoters, the viral MND promoter and the human EF1α promoter. In vivo studies showed stable and persistent GFP expression throughout the cell bodies, axons, and dendrites from intracortical injections and indicated ependymal and subependymal transduction. The sheep showed no ill effects from the injections. These data support continuing gene-therapy trials in the sheep models of Batten disease.
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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.