Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models.

@article{Pellissier2015GeneTI,
  title={Gene therapy into photoreceptors and M{\"u}ller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models.},
  author={Lucie P Pellissier and Peter M. Quinn and Celso Henrique Alves and Rogier M. Vos and J. van't Klooster and John G Flannery and J Alexander Heimel and Jan Wijnholds},
  journal={Human molecular genetics},
  year={2015},
  volume={24 11},
  pages={3104-18}
}
Mutations in the Crumbs-homologue-1 (CRB1) gene lead to severe recessive inherited retinal dystrophies. Gene transfer therapy is the most promising cure for retinal dystrophies and has primarily been applied for recessive null conditions via a viral gene expression vector transferring a cDNA encoding an enzyme or channel protein, and targeting expression to one cell type. Therapy for the human CRB1 disease will be more complex, as CRB1 is a structural and signaling transmembrane protein present… CONTINUE READING

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