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Isolation of Primary Mouse Retinal Glial Müller Cells
Published on: August 30, 2024
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Specific tools for targeting and expression in Müller glial cells.
Lucie P Pellissier1, Robert M Hoek1, Rogier M Vos1
1Department of Neuromedical Genetics, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
Molecular Therapy. Methods & Clinical Development
|May 28, 2015
Summary
This study identifies effective adeno-associated virus (AAV) capsids (ShH10Y, AAV9) and promoters (RLBP1, minimal CMV) for targeting Müller glial cells. These tools are promising for treating retinal diseases like CRB1 inherited retinal dystrophies.
Area of Science:
- Ophthalmology
- Neuroscience
- Gene Therapy
Background:
- Müller glial cells play crucial roles in retinal physiology.
- These cells are implicated in retinal disease pathogenesis and regeneration.
- Targeting Müller glial cells offers therapeutic potential for inherited retinal dystrophies.
Purpose of the Study:
- To evaluate adeno-associated virus (AAV) capsid variants and promoters for Müller glial cell transduction.
- To identify efficient tools for gene expression in Müller glial cells for therapeutic applications.
- To develop methods for expressing large genes, such as CRB1, in Müller glial cells.
Main Methods:
- Screening of AAV capsid variants (ShH10Y, AAV9) for Müller glial cell infection efficiency in mice.
- Testing various promoters, including Retinaldehyde-binding protein 1 (RLBP1) and minimal Cytomegalovirus (CMV), for Müller glial cell transduction.
- In vitro assessment of ShH10Y capsids and RLBP1 promoter in human Müller glial cells.
- Development and testing of smaller promoters for expressing the CRB1 gene via AAV vectors.
Main Results:
- ShH10Y and AAV9 capsids demonstrated high efficiency in infecting mouse Müller glial cells.
- The RLBP1 promoter was the most effective for transducing Müller glial cells.
- ShH10Y capsids and RLBP1 promoter showed efficacy in targeting human Müller glial cells in vitro.
- A minimal CMV promoter enabled the expression of the full-length CRB1 protein in Müller glial cells.
Conclusions:
- ShH10Y and AAV9 capsids are potent tools for targeting mouse Müller glial cells.
- RLBP1 and minimal CMV promoters are effective for Müller glial cell-specific gene expression.
- These identified AAV capsids and promoters are valuable for developing gene therapies for retinal diseases affecting Müller glial cells, including CRB1 inherited retinal dystrophies.
