ArticleHuman gene therapy methods2012
Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation.
Article in Human gene therapy methods, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
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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
28 citing papers in PubMed, 35 citations in OpenAlex.
- Improving AAV Production Yield and Quality for Different Serotypes Using Distinct Processing Methods.ACS omega · 2025Article
- An SDS-NaOH-based method to isolate genome of recombinant adeno-associated virus vectors for physical titer measurement.PloS one · 2025Article
- Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.Molecular therapy. Methods & clinical development · 2023Article
- PCR-Based Analytical Methods for Quantification and Quality Control of Recombinant Adeno-Associated Viral Vector Preparations.Pharmaceuticals (Basel, Switzerland) · 2021Review
- Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies.Molecular therapy. Methods & clinical development · 2021Review
- Adeno-Associated Virus Vector Mobilization, Risk Versus Reality.Human gene therapy · 2020Article
- Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- A Novel Next-Generation Sequencing and Analysis Platform to Assess the Identity of Recombinant Adeno-Associated Viral Preparations from Viral DNA Extracts.Human gene therapy · 2020Article
- Quantitative and Digital Droplet-Based AAV Genome Titration.Methods in molecular biology (Clifton, N.J.) · 2019Article
- Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.Neural plasticity · 2018Review
- Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- The hepatocyte-specific HNF4α/miR-122 pathway contributes to iron overload-mediated hepatic inflammation.Blood · 2017Article
- Deletion of the B-B' and C-C' regions of inverted terminal repeats reduces rAAV productivity but increases transgene expression.Scientific reports · 2017Article
- Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders.Gene therapy · 2017Article
- Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration.Current stem cell reports · 2017Review
- Microglia-specific targeting by novel capsid-modified AAV6 vectors.Molecular therapy. Methods & clinical development · 2016Article
- Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors.Molecular therapy. Methods & clinical development · 2016Article
- Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing.Molecular therapy. Nucleic acids · 2015Article
- Productive life cycle of adeno-associated virus serotype 2 in the complete absence of a conventional polyadenylation signal.The Journal of general virology · 2015Article
- Neuritin reverses deficits in murine novel object associative recognition memory caused by exposure to extremely low-frequency (50 Hz) electromagnetic fields.Scientific reports · 2015Article
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 2 countries.
Funding
Abstract
We previously reported that self-complementary adeno-associated virus (scAAV) type 2 genomes of up to 3.3 kb can be successfully encapsidated into AAV2 serotype capsids. Here we report that such oversized AAV2 genomes fail to undergo packaging in other AAV serotype capsids, such as AAV1, AAV3, AAV6, and AAV8, as determined by Southern blot analyses of the vector genomes, although hybridization signals on quantitative DNA slot-blots could still be obtained. Recently, it has been reported that quantitative real-time PCR assays may result in substantial differences in determining titers of scAAV vectors depending on the distance between the primer sets and the terminal hairpin structure in the scAAV genomes. We also observed that the vector titers determined by the standard DNA slot-blot assays were highly dependent on the specific probe being used, with probes hybridizing to the ends of viral genomes being significantly overrepresented compared with the probes hybridizing close to the middle of the viral genomes. These differences among various probes were not observed using Southern blot assays. This overestimation of titer is a systemic error during scAAV genome quantification, regardless of viral genome sequences and capsid serotypes. Furthermore, different serotypes capsid and modification of capsid sequence may affect the ability of packaging intact, full-length AAV genomes. Although the discrepancy is modest with wild-type serotype capsid and short viral genomes, the measured titer could be as much as fivefold different with capsid mutant vectors and large genomes. Thus, based on our data, we suggest that Southern blot analyses should be performed routinely to more accurately determine the titers of recombinant AAV vectors. At the very least, the use of probes/primers hybridizing close to the mutant inverted terminal repeat in scAAV genomes is recommended to avoid possible overestimation of vector titers.
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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.