ArticlePediatric research2015
Augmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses.
Article in Pediatric research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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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
10 citing papers in PubMed, 10 citations in OpenAlex.
- Towards enhancing photostability and adeno-associated viral vector delivery for genetically encoded plasma fluorescent labels.Neurophotonics · 2025Article
- Preclinical Milestones in MECP2 Gene Transfer for Treating Rett Syndrome.Developmental neuroscience · 2025Review
- The Efficacy of a Human-Ready miniGenes · 2023Article
- Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice.Nature medicine · 2023Article
- In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.Nature communications · 2022Article
- Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency.Molecular therapy. Nucleic acids · 2022Article
- Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy.Molecular genetics and metabolism · 2018Article
- miR-29b supplementation decreases expression of matrix proteins and improves alveolarization in mice exposed to maternal inflammation and neonatal hyperoxia.American journal of physiology. Lung cellular and molecular physiology · 2017Article
- Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2016Article
- DHA suppresses chronic apoptosis in the lung caused by perinatal inflammation.American journal of physiology. Lung cellular and molecular physiology · 2015Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
backgroundAchieving persistent expression is a prerequisite for genetic therapies for inherited metabolic enzymopathies. Such disorders potentially could be treated with gene therapy shortly after birth to prevent pathology. However, rapid cell turnover leads to hepatic episomal vector loss, which diminishes effectiveness. The current studies assessed whether tolerance to transgene proteins expressed in the neonatal period is durable and if the expression may be augmented with subsequent adeno-associated virus (AAV) administration.
methodsAAV was administered to mice on day 2 with reinjection at 14 or at 14 and 42 d with examination of changes in hepatic copies and B and T cell-mediated immune responses.
resultsImmune responses to the transgene protein and AAV were absent after neonatal administration. Reinjection at 14 or at 14 and 42 d resulted in augmented expression with greater hepatic genome copies. Unlike controls, immune responses to transgene proteins were not detected in animals injected as neonates and subsequently. However, while no immune response developed after neonatal administration, anticapsid immune responses developed with further injections suggesting immunological ignorance was the initial mechanism of unresponsiveness.
conclusionsPersistence of transgene protein allows for tolerance induction permitting readministration of AAV to re-establish protein levels that decline with growth.
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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.