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Control of Microbial Opsin Expression in Stem Cell Derived Cones for Improved Outcomes in Cell Therapy
Marcela Garita-Hernandez1, Antoine Chaffiol1, Laure Guibbal1
1Institut de la Vision, Sorbonne Université, Paris, France.
Frontiers in Cellular Neuroscience
|April 5, 2021
Summary
Human organoids derived from pluripotent stem cells are valuable for modeling disease and cell therapy. This study introduces a specific promoter to control gene expression in retinal organoids, improving therapeutic potential.
Area of Science:
- Stem cell biology
- Ophthalmology
- Gene therapy
Background:
- Human-induced pluripotent stem cell (hiPSC)-derived organoids offer 3D models for human organ development and disease.
- These organoids are crucial for cell therapy and gene therapy validation.
- Genetic modification tools are essential for controlling organoid function in various applications.
Purpose of the Study:
- To develop and validate a genetic tool for precise control of gene expression in hiPSC-derived retinal organoids.
- To enhance gene expression specifically in cone cells using a novel promoter system.
- To improve the therapeutic efficacy of cell transplantation by optimizing transgene expression.
Main Methods:
- Utilized adeno-associated virus (AAV) vectors for gene delivery into human retinal organoids.
- Employed a 1.7-kb L-opsin promoter for cell-type-specific gene expression in cone cells.
- Fused the red-shifted opsin Jaws with a fluorescent reporter gene for enhanced expression and cell sorting.
- Assessed Jaws expression and functional light responses after transplantation.
Main Results:
- Demonstrated biological restriction and enhanced gene expression in retinal organoid cone cells using the L-opsin promoter.
- Successfully enriched desired cell populations through cell sorting based on fluorescent reporter expression.
- Observed improved light responses in transplanted cells with increased Jaws expression.
- Showcased the promoter's ability to control the kinetics of transgene expression during organoid maturation.
Conclusions:
- Cell-type-specific promoters are critical for controlling transgene expression during the differentiation and maturation of hiPSC retinal derivatives.
- Implementing promoters like the L-opsin promoter enhances gene expression and refines transcriptional control in mature cell states.
- This approach holds significant promise for advancing cell therapy and gene therapy in ophthalmology.
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