ArticleThe journal of gene medicine2010
RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy.
Article in The journal of gene medicine, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 papers.
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Who cites it
54 citing papers in PubMed, 78 citations in OpenAlex.
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- 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
- Gene Therapy for Acquired and Genetic Cholestasis.Biomedicines · 2022Review
- Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery.Scientific reports · 2021Article
- Long-term functional correction of cystathionine β-synthase deficiency in mice by adeno-associated viral gene therapy.Journal of inherited metabolic disease · 2021Article
- Overexpression of human BAG3Nature communications · 2021Article
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- High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models.Molecular therapy. Methods & clinical development · 2020Article
- High-ThroughputHuman gene therapy · 2020Article
- Comparative Analysis of the Capsid Structures of AAVrh.10, AAVrh.39, and AAV8.Journal of virology · 2020Article
- Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model.Nature communications · 2019Article
- Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- rAAVrh74.MCK.GALGT2 Protects against Loss of Hemodynamic Function in the Aging mdx Mouse Heart.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Adeno-Associated Virus-Mediated Gene Transfer of Tissue Inhibitor of Metalloproteinases-1 Impairs Neutrophil Extracellular Trap Formation and Ameliorates Hepatic Ischemia and Reperfusion Injury.The American journal of pathology · 2018Article
- Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice.Molecular genetics and metabolism · 2018Article
- Adeno-associated virus serotype rh10 is a useful gene transfer vector for sensory nerves that innervate bone in immunodeficient mice.Scientific reports · 2017Article
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
backgroundNeonatal gene therapy is a promising strategy for treating diseases diagnosed before or shortly after birth. Early and long-term expression of therapeutic proteins may limit the consequences of genetic mutations and result in a potential 'cure'. Adeno-associated viral vectors have shown promise in many areas of adult gene therapy but their properties have not been systematically investigated in the neonate.
methodsIn these studies, using a constitutive promoter expressing luciferase, animals were administered one of ten serotypes of adeno-associated virus (AAV) on the second day of life. Examination of expression, organ growth and vector distribution, maintenance of expression and copy number were measured.
resultsAll serotypes demonstrated expression and, in general, transduction of all organs within 3 days, albeit with different biodistribution patterns and expression levels. The highest expression was detected with AAVrh10, whereas the lowest was detected with AAV4. Expression and genomes declined with growth over the first 10 weeks of life; thereafter, to day 100, expression and genomes remained relatively stable. With the highest expressing vectors, whole animal expression at 100 days declined to approximately 10% of that detected on the fifth day. AAVrh10 maintained the highest expression level and copy number throughout these studies.
conclusionsThe impact of tissue and organ growth on the stability of AAV expression will be important if neonatal gene transfer is to be considered as a modality for human gene therapy. Although all vectors did demonstrate expression, rh10 holds the greater promise of the vectors tested to maintain copy number in both mitotic and post-mitotic tissues.
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