Journal Club | Developing Retinal Gene Therapy for Zellweger Spectrum Disorder (ZSD)
Автор: Striatech GmbH
Загружено: 2023-08-15
Просмотров: 449
Описание:
Catherine Argyriou, PhD - McGill University, Dept of Human Genetics
The presentation will provide background on peroxisomal disorders and the PEX1-G844D mouse model for mild Zellweger Spectrum Disorder, which presents with retinal degeneration and vision loss. The presenter will describe how her team used AAV8-mediated gene delivery to robustly and durably improve functional vision, retinal response, retinal structure, and biochemical metabolites in ZSD mice. Techniques used include subretinal injection, ERG, optomotor testing, histology, immunohistochemistry, and LC-MSMS. AAV vector design and validation will also be discussed. This work attracted venture capital investment and is now ready to enter the clinical translation phase.
See our Website for more information: https://stria.tech/journal-club-retin...
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