ArticleHuman gene therapy methods2015
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.
Article in Human gene therapy methods, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed.
- A murine model of amyloid-enhanced tauopathy: Comparison of wild-type and P301L-tau.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026Article
- Reactive species generated by a cold atmospheric plasma inactivate AAV through oxidative and nitrosative stress.Scientific reports · 2026Article
- Assessing the biopotency of the rAAV9 vector In Vitro.PloS one · 2026Article
- A Review on the Stability Challenges of Advanced Biologic Therapeutics.Pharmaceutics · 2025Review
- Physicochemical and biological impacts of light stress on adeno-associated virus serotype 6.Molecular therapy. Methods & clinical development · 2024Article
- Characterization of AAV vectors: A review of analytical techniques and critical quality attributes.Molecular therapy. Methods & clinical development · 2024Review
- An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species.Nature communications · 2024Article
- Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF.BMC biotechnology · 2024Article
- Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.Molecular therapy. Methods & clinical development · 2023Article
- Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate.Molecular therapy. Methods & clinical development · 2023Article
- Overcoming Barriers to Preventing and TreatingBiomedicines · 2022Review
- Optimized formulation buffer preserves adeno-associated virus-9 infectivity after 4 °C storage and freeze/thawing cycling.Journal of virological methods · 2022Article
- AAV8 transduction capacity is reduced by prior exposure to endosome-like pH conditions.Neural regeneration research · 2021Article
- Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?Frontiers in immunology · 2021Review
- Effects of Thermally Induced Configuration Changes on rAAV Genome's Enzymatic Accessibility.Molecular therapy. Methods & clinical development · 2020Article
- Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth.Frontiers in immunology · 2020Article
- Chimeric Capsid Proteins Impact Transduction Efficiency of Haploid Adeno-Associated Virus Vectors.Viruses · 2019Article
- Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein.Journal of virology · 2019Article
- AAV Vectors for Efficient Gene Delivery to Rodent Hearts.Methods in molecular biology (Clifton, N.J.) · 2019Article
- Pharmacology of Recombinant Adeno-associated Virus Production.Molecular therapy. Methods & clinical development · 2018Review
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
Recombinant adeno-associated virus (rAAV) vectors are rapidly becoming the first choice for human gene therapy studies, as clinical efficacy has been demonstrated in several human trials and proof-of-concept data have been demonstrated for correction of many others. When moving into human use under the auspices of an FDA Investigational New Drug (IND) application, it is necessary to demonstrate the stability of vector material under various conditions of storage, dilution, and administration when used in humans. Limited data are currently available in the literature regarding vector compatibility and stability, leading most IND sponsors to repeat all necessary studies. The current study addresses this issue with an rAAV vector (rAAV1-CB-chAATmyc) containing AAV2-inverted terminal repeat sequences packaged into an AAV1 capsid. Aliquots of vector were exposed to a variety of temperatures, diluents, container constituents, and other environmental conditions, and its functional biological activity (after these various treatments) was assessed by measuring transgene expression after intramuscular injection in mice. rAAV was found to be remarkably stable at temperatures ranging from 4°C to 55°C (with only partial loss of potency after 20 min at 70°C), at pH ranging from 5.5 to 8.5, after contact with mouse or human serum (with or without complement depletion) or with gadolinium and after contact with glass, polystyrene, polyethylene, polypropylene, and stainless steel. The only exposure resulting in near-total loss of vector activity (10,000-fold loss) was UV exposure for 10 min. The stability of rAAV1 preparations bodes well for future dissemination of this therapeutic modality.
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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.