ArticleHuman gene therapy2017
Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.
Article in Human gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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
16 citing papers in PubMed, 33 citations in OpenAlex.
- Monitoring biological effects of somatic cell genome editing.Nature reviews. Genetics · 2026Review
- Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects.Gene therapy · 2024Article
- Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- Engineered nanoparticles promote cardiac tropism of AAV vectors.Journal of nanobiotechnology · 2024Article
- Redirecting AAV vectors to extrahepatic tissues.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery.Nature protocols · 2023Review
- Context-Specific Function of the Engineered Peptide Domain of PHP.B.Journal of virology · 2021Article
- A brief review of reporter gene imaging in oncolytic virotherapy and gene therapy.Molecular therapy oncolytics · 2021Review
- The NIH Somatic Cell Genome Editing program.Nature · 2021Review
- Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back.Journal of personalized medicine · 2020Review
- Dosing and Re-Administration of Lentiviral Vector forMolecular therapy. Methods & clinical development · 2020Article
- In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery.Scientific reports · 2020Article
- AAVR-Displaying Interfaces: Serotype-Independent Adeno-Associated Virus Capture and Local Delivery Systems.Molecular therapy. Nucleic acids · 2019Article
- Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys.Human gene therapy · 2019Article
- Adeno-Associated Virus (AAV) as a Vector for Gene Therapy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2017Review
- Site-Specific PEGylated Adeno-Associated Viruses with Increased Serum Stability and Reduced Immunogenicity.Molecules (Basel, Switzerland) · 2017Article
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5 authors at 2 institutions in 1 country.
Funding
Abstract
The liver is a major off-target organ in gene therapy approaches for cardiac and musculoskeletal disorders. Intravenous administration of most of the naturally occurring adeno-associated virus (AAV) strains invariably results in vector genome sequestration within the liver. In the current study, we compared the muscle tropism and transduction efficiency of a liver de-targeted AAV variant to AAV9 following systemic administration in newborn rhesus monkeys. In vivo bioluminescence imaging was performed to monitor transgene expression (firefly luciferase) post administration. Results indicated comparable and sustained levels of systemic firefly luciferase gene expression in skeletal muscle over a period of two years. Quantitation of vector biodistribution in harvested tissues post-administration revealed widespread recovery of vector genomes delivered by AAV9 but markedly decreased levels in major systemic organs from the AAV variant. These studies validate the translational potential and safety of liver de-targeted AAV strains for gene therapy of muscle-related diseases.
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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.