ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 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.
The trial behind it
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Who cites it
24 citing papers in PubMed, 38 citations in OpenAlex.
- Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates.Gene therapy · 2022Article
- Article
- The Dog Model in the Spotlight: Legacy of a Trustful Cooperation.Journal of neuromuscular diseases · 2019Article
- Gene therapy for ocular diseases meditated by ultrasound and microbubbles (Review).Molecular medicine reports · 2015Review
- Fibrogenic Cell Plasticity Blunts Tissue Regeneration and Aggravates Muscular Dystrophy.Stem cell reports · 2015Article
- Duchenne muscular dystrophy gene therapy in the canine model.Human gene therapy. Clinical development · 2015Review
- Elimination of contaminating cap genes in AAV vector virions reduces immune responses and improves transgene expression in a canine gene therapy model.Gene therapy · 2014Article
- Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.Pharmaceuticals (Basel, Switzerland) · 2013Article
- Gene therapy for cardiovascular disease mediated by ultrasound and microbubbles.Cardiovascular ultrasound · 2013Review
- Injection of vessel-derived stem cells prevents dilated cardiomyopathy and promotes angiogenesis and endogenous cardiac stem cell proliferation in mdx/utrn-/- but not aged mdx mouse models for duchenne muscular dystrophy.Stem cells translational medicine · 2013Article
- Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Immunosuppression decreases inflammation and increases AAV6-hSERCA2a-mediated SERCA2a expression.Human gene therapy · 2012Article
- Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.Human molecular genetics · 2012Article
- Gene-based passive antibody protection from HIV.Nature biotechnology · 2012Article
- Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies.Mammalian genome : official journal of the International Mammalian Genome Society · 2012Review
- Transduction of skeletal muscles with common reporter genes can promote muscle fiber degeneration and inflammation.PloS one · 2012Article
- Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- Therapeutic approaches to muscular dystrophy.Human molecular genetics · 2011Review
Corrections and comments
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Authors and funding
11 authors at 2 institutions in 1 country.
Funding
Abstract
We previously demonstrated that direct intramuscular injection of rAAV2 or rAAV6 in wild-type dogs resulted in robust T-cell responses to viral capsid proteins, and others have shown that cellular immunity to adeno-associated virus (AAV) capsid proteins coincided with liver toxicity and elimination of transgene expression in a human trial of hemophilia B. Here, we show that the heparin-binding ability of a given AAV serotype does not determine the induction of T-cell responses following intramuscular injection in dogs, and identify multiple epitopes in the AAV capsid protein that are recognized by T cells elicited by AAV injection. We also demonstrate that noninvasive magnetic resonance imaging (MRI) can accurately detect local inflammatory responses following intramuscular rAAV injection in dogs. These studies suggest that pseudotyping rAAV vectors to remove heparin-binding activity will not be sufficient to abrogate immunogenicity, and validate the utility of enzyme-linked immunosorbent spot (ELISpot) assay and MRI for monitoring immune and inflammatory responses following intramuscular injection of rAAV vectors in preclinical studies in dogs. These assays should be incorporated into future human clinical trials of AAV gene therapy to monitor immune responses.
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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.