ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.
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 105 papers.
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Who cites it
105 citing papers in PubMed, 161 citations in OpenAlex.
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- Peptide ligands for the affinity purification of adeno-associated viruses from HEK 293 cell lysates.Biotechnology and bioengineering · 2023Article
- Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model.Human gene therapy · 2023Article
- Progress in Clinical Gene Therapy for Cardiac Disorders.Molecular diagnosis & therapy · 2023Review
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- Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy.Molecular therapy. Methods & clinical development · 2022Article
- Rational engineering of a functional CpG-free ITR for AAV gene therapy.Gene therapy · 2022Article
- Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers.Molecular therapy. Methods & clinical development · 2022Article
- Programmable RNA-Guided Large DNA Transgenesis by CRISPR/Cas9 and Site-Specific Integrase Bxb1.Frontiers in bioengineering and biotechnology · 2022Review
- Adeno-associated virus-mediated expression of activated factor V (FVa) for hemophilia phenotypic correction.Frontiers in medicine · 2022Article
- Strategies for Targeting Neural Circuits: How to Manipulate Neurons Using Virus Vehicles.Frontiers in neural circuits · 2022Review
- Gene Therapy in the Anterior Eye Segment.Current gene therapy · 2022Review
- Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry.Molecular therapy. Methods & clinical development · 2021Article
45 more citing papers are in PubMed but not listed here.
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3 authors at 1 institution in 1 country.
Funding
Abstract
Limited packaging capacity hinders adeno-associated virus (AAV) gene therapy. A recent study seems to have provided a solution to this problem. Allocca et al. reported that AAV-5 could package an 8.9 kb vector genome. Here we tested whether this approach can be used to deliver a large genome for Duchenne muscular dystrophy (DMD) gene therapy. We first evaluated AAV-5 packaging of an 8.2 kb genome. This vector carries two independent reporter gene cassettes, one for alkaline phosphatase (AP) and another for LacZ. Viral yield was log-fold lower than that of a regular AAV-5. Nevertheless, both AP and LacZ genes were detected in purified virus. Injection to dystrophic muscle resulted in both AP and LacZ expression. On electron microscopy, virion structure appeared normal. Surprisingly, we did not find the full-length single-stranded viral genome by alkaline gel electrophoresis. Neither did we see the full-length double-stranded replication forms in adenovirus coinfected cells. We suspect that AP and LacZ expression may have come from partially packaged 5' or 3'-half of the genome. Additional studies revealed failure of AAV-5 to package and express an 8.7 kb minidystrophin gene cassette. In summary, our results do not support the extraordinary packaging capacity of AAV-5.
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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.