title: Specific tools for targeting and expression in Müller glial cells.
abstract: Despite their physiological roles, Müller glial cells are involved directly or indirectly in retinal disease pathogenesis and are an interesting target for therapeutic approaches for retinal diseases and regeneration such as CRB1 inherited retinal dystrophies. In this study, we characterized the efficiency of adeno-associated virus (AAV) capsid variants and different promoters to drive protein expression in Müller glial cells. ShH10Y and AAV9 were the most powerful capsids to infect mouse Müller glial cells. Retinaldehyde-binding protein 1 (RLBP1) promoter was the most powerful promoter to transduce Müller glial cells. ShH10Y capsids and RLBP1 promoter targeted human Müller glial cells in vitro. We also developed and tested smaller promoters to express the large CRB1 gene via AAV vectors. Minimal cytomegalovirus (CMV) promoter allowed expression of full-length CRB1 protein in Müller glial cells. In summary, ShH10Y and AAV9 capsids, and RLBP1 or minimal CMV promoters are of interest as specific tools to target and express in mouse or human Müller glial cells.
authors.affiliation: Department of Neuromedical Genetics, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
authors.forename: Lucie P
authors.initials: LP
authors.lastname: Pellissier
authors.affiliation: Department of Neuromedical Genetics, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
authors.forename: Robert M
authors.initials: RM
authors.lastname: Hoek
authors.affiliation: Department of Neuromedical Genetics, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
authors.forename: Rogier M
authors.initials: RM
authors.lastname: Vos
authors.affiliation: Department of Neuromedical Genetics, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
authors.forename: Wendy M
authors.initials: WM
authors.lastname: Aartsen
authors.affiliation: Department of Molecular and Cellular Biology and The Helen Wills Neuroscience Institute, University of California , Berkeley, California, USA.
authors.forename: Ryan R
authors.initials: RR
authors.lastname: Klimczak
authors.affiliation: Department of Neuroregeneration, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
authors.forename: Stefan A
authors.initials: SA
authors.lastname: Hoyng
authors.affiliation: Department of Molecular and Cellular Biology and The Helen Wills Neuroscience Institute, University of California , Berkeley, California, USA.
authors.forename: John G
authors.initials: JG
authors.lastname: Flannery
authors.affiliation: Department of Neuromedical Genetics, Netherlands Institute for Neuroscience, an institute of the Royal Netherlands Academy of Arts and Sciences , Amsterdam, The Netherlands.
authors.forename: Jan
authors.initials: J
authors.lastname: Wijnholds