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Updated: Aug 5, 2026
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Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease
Published on: April 19, 2015
Storage and Stability of AAV-Containing Fibrin Hydrogels for Retinal Gene Therapy
Aubrey Berger1,2, Travis Knudsen2, Francesca Kopp2
1Biomedical Graduate Program, Mayo Clinic, Rochester, MN 55905, USA.
Gels (Basel, Switzerland)
|July 27, 2026
Summary
Fibrin hydrogel encapsulated adeno-associated virus (FE-AAV) offers improved retinal gene therapy delivery. Formulating FE-AAV with Pluronic F68 and storing at 4°C ensures stability and efficacy for at least 4 months.
Area of Science:
- Ophthalmology
- Gene Therapy
- Biomaterials Science
Background:
- Current subretinal and intravitreal injection methods for retinal gene therapy face challenges with adverse events and limited efficacy.
- Developing novel delivery systems is crucial for advancing retinal gene therapy applications.
- Adeno-associated virus (AAV) is a common vector for gene delivery, but its stability and effective delivery to target retinal cells require optimization.
Purpose of the Study:
- To develop and optimize fibrin hydrogel encapsulated adeno-associated virus (FE-AAV) for improved retinal gene therapy delivery.
- To investigate the storage conditions and stability of FE-AAV, focusing on formulation and temperature.
- To assess the impact of different formulations and storage durations on FE-AAV's physical, mechanical, and transduction properties.
Main Methods:
- FE-AAV was manufactured using fibrinogen reconstituted in either sodium citrate (NaC) or phosphate-buffered saline with Pluronic F68 (F68).
- Samples were stored at -80°C or 4°C for up to 16 weeks.
- Transduction efficiency, and mechanical and physical properties were evaluated post-storage.
Main Results:
- FE-AAV manufactured with NaC showed significantly reduced in vitro transduction efficiency (p < 0.05).
- FE-AAV formulated with F68 maintained consistent transduction efficiency throughout the 16-week storage period.
- Physical changes were observed in FE-AAV stored at -80°C, whereas FE-AAV formulated with F68 and stored at 4°C remained equivalent to fresh preparations, retaining RPE transduction capability.
Conclusions:
- FE-AAV formulated with Pluronic F68 demonstrates superior stability compared to NaC formulation.
- Storage at 4°C is optimal for maintaining FE-AAV integrity and function for extended periods (at least 4 months).
- This optimized FE-AAV formulation shows significant potential as a stable and effective delivery system for retinal gene therapy.
