ArticleThe Journal of clinical investigation2006
Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia.
Article in The Journal of clinical investigation, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
22 citing papers in PubMed, 42 citations in OpenAlex.
- Pluripotent Stem Cells as a Preclinical Cellular Model for Studying Hereditary Spastic Paraplegias.International journal of molecular sciences · 2024Review
- Pharmacological rescue of mitochondrial and neuronal defects inFrontiers in neuroscience · 2023Article
- Hereditary Spastic Paraplegia: An Update.International journal of molecular sciences · 2022Review
- Altered Mitochondrial Protein Homeostasis and Proteinopathies.Frontiers in molecular neuroscience · 2022Review
- Hereditary spastic paraplegia.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2021Review
- Insights into Clinical, Genetic, and Pathological Aspects of Hereditary Spastic Paraplegias: A Comprehensive Overview.Frontiers in molecular biosciences · 2021Review
- A Single Intravenous Injection of AAV-PHP.B-Molecular therapy. Methods & clinical development · 2020Article
- Complexity of Generating Mouse Models to Study the Upper Motor Neurons: Let Us Shift Focus from Mice to Neurons.International journal of molecular sciences · 2019Review
- Genetic manipulation of autonomic nerve fiber innervation and activity and its effect on breast cancer progression.Nature neuroscience · 2019Article
- Update on the Genetics of Spastic Paraplegias.Current neurology and neuroscience reports · 2019Review
- Management of Hereditary Spastic Paraplegia: A Systematic Review of the Literature.Frontiers in neurology · 2019Article
- Loss of the Drosophila m-AAA mitochondrial protease paraplegin results in mitochondrial dysfunction, shortened lifespan, and neuronal and muscular degeneration.Cell death & disease · 2018Article
- Clinical Trial Designs and Measures in Hereditary Spastic Paraplegias.Frontiers in neurology · 2018Review
- Expanded phenotype in a patient with spastic paraplegia 7.Clinical case reports · 2017Article
- An Update on the Hereditary Spastic Paraplegias: New Genes and New Disease Models.Movement disorders clinical practice · 2015Review
- A spastic paraplegia mouse model reveals REEP1-dependent ER shaping.The Journal of clinical investigation · 2013Article
- Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.Human gene therapy methods · 2012Article
- Article
- Efficient retrograde transport of adeno-associated virus type 8 to spinal cord and dorsal root ganglion after vector delivery in muscle.Human gene therapy · 2010Article
- Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6.Gene therapy · 2010Article
Corrections and comments
- Erratum issued
Authors and funding
7 authors at 5 institutions in 1 country.
Funding
Abstract
Degeneration of peripheral motor axons is a common feature of several debilitating diseases including complicated forms of hereditary spastic paraplegia. One such form is caused by loss of the mitochondrial energy-dependent protease paraplegin. Paraplegin-deficient mice display a progressive degeneration in several axonal tracts, characterized by the accumulation of morphological abnormal mitochondria. We show that adenoassociated virus-mediated (AAV-mediated) intramuscular delivery of paraplegin halted the progression of neuropathological changes and rescued mitochondrial morphology in the peripheral nerves of paraplegin-deficient mice. One single injection before onset of symptoms improved the motor performance of paraplegin-deficient mice for up to 10 months, indicating that the peripheral neuropathy contributes to the clinical phenotype. This study provides a proof of principle that gene transfer may be an effective therapeutic option for patients with paraplegin deficiency and demonstrates that AAV vectors can be successfully employed for retrograde delivery of an intracellular protein to spinal motor neurons, opening new perspectives for several hereditary axonal neuropathies of the peripheral nerves.
Indexed as
Identifiers
What OpenQuestion holds
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.