ArticleMolecular therapy. Methods & clinical development2020
Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy.
Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed.
- Mechanistic Insights into Poloxamer 188-Mediated Stabilization of AAV8 Capsids Using Coarse-Grained Molecular Dynamics.Pharmaceutical research · 2026Article
- Storage and Stability of AAV-Containing Fibrin Hydrogels for Retinal Gene Therapy.Gels (Basel, Switzerland) · 2026Article
- Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF.BMC biotechnology · 2024Article
- Retention Rates of Genetic Therapies Based on AAV Serotypes 2 and 8 Using Different Drug-Delivery Materials.International journal of molecular sciences · 2024Article
- Combined clarification and affinity capture using magnetic resin enables efficient separation of rAAV5 from cell lysate.Molecular therapy. Methods & clinical development · 2023Article
- Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl.Cold Spring Harbor perspectives in medicine · 2023Review
- High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography.Molecular therapy. Methods & clinical development · 2023Article
- Intravitreal Injection of AAV for the Transduction of Mouse Retinal Ganglion Cells.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Genome concentration, characterization, and integrity analysis of recombinant adeno-associated viral vectors using droplet digital PCR.PloS one · 2023Article
- Thermostability and in vivo performance of AAV9 in a film matrix.Communications medicine · 2022Article
- Optimized formulation buffer preserves adeno-associated virus-9 infectivity after 4 °C storage and freeze/thawing cycling.Journal of virological methods · 2022Article
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Authors and funding
6 authors.
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Abstract
Recent advances in recombinant adeno-associated virus (rAAV) gene therapy for choroideremia show gene replacement to be a promising approach. It is, however, well known that contact of vector solution with plastic materials in the surgical device may result in non-specific adsorption with resulting loss of physical titer and/or level of protein expression and activity. Here we assessed the biocompatibility and stability of rAAV2-REP1 (Rab Escort Protein-1) before and following passage through the injection device over a period of time to mimic the clinical scenario. Three identical devices were screened using two concentrations of vector: high (1E+12 DNase-resistant particles [DRP]/mL) and low (1E+11 DRP/mL), to mimic high- and low-dose administrations of vector product. The low dose was prepared using either formulation buffer that contained 0.001% of a non-ionic surfactant (PF68) or balanced salt solution (BSS). We observed significant losses in the genomic titer of samples diluted with BSS for all time points. The addition of 0.001% PF68 did not, however, affect rAAV physical titer, or REP1 protein expression and biological activity. Hence we observed that neither the genomic titer nor the biological activity of a rAAV2-REP1-containing solution was affected following passage through the surgical device when PF68 was present as a surfactant and this was maintained over a period up to 10 h.
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