Glycosidic Enzymes Improve Retinal Gene Therapy
Author Information
Author(s): Cehajic-Kapetanovic Jasmina, Le Goff Magali M., Allen Annette, Lucas Robert J., Bishop Paul N.
Primary Institution: University of Manchester
Hypothesis
Can the co-injection of extracellular matrix degrading enzymes improve retinal transduction following intravitreal delivery of AAV2?
Conclusion
Co-injection of heparinase III or chondroitin ABC lyase significantly enhances AAV2-mediated retinal transduction.
Supporting Evidence
- The addition of heparinase III or chondroitin ABC lyase greatly enhanced transduction efficiency.
- Electroretinograms showed preserved retinal function after enzyme treatment.
- Collagenase was ineffective in enhancing transduction.
- Hyaluronan lyase had a limited effect on transduction.
Takeaway
This study found that adding certain enzymes when delivering a gene therapy to the eye helps the treatment work better.
Methodology
Adult mice received intravitreal injections of AAV2 with various enzymes, and retinal transduction was assessed using confocal microscopy and electroretinograms.
Limitations
The study primarily used mouse models, which may not fully represent human responses.
Participant Demographics
Adult Opn4-/+ and wild-type C57BL/6J mice were used.
Statistical Information
P-Value
p<0.05
Confidence Interval
95% CI
Statistical Significance
p<0.05
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