The Retinal Crumbs Complex: from animal models and retinal organoids to therapy

Author(s):
Quinn, P.M.J.
Published
2019
Publication type
Doctoral Thesis

Abstract

By investigating the roles of CRB proteins in mouse, non-human-primate, human fetal retina, and iPSC-derived retinal organoids, this thesis describes important insights to pathobiology in CRB1-retinitis pigmentosa (RP) and CRB1-Leber congenital amaurosis (LCA) disease models. The thesis describes AAV gene and cell therapy-based tools as therapeutic strategies for alleviation of RP and LCA due to loss of CRB1.

CRB1Apical PolarityLeber congenital amaurosisRetinitis pigmentosaGene therapyRetinaRetinal organoids
Defense date
2019-05-15
Language
English
Supervisor(s)
Luyten, G.P.M.
Advisor(s)
Wijnholds, J.
Committee member(s)
Bergen, A.A.B.; Verhaagen, J.; Boon, C.J.F.; Baker, D.A.; Weerd, L. van der
Qualification
Ph.D.
Degree granting institution
Ophthalmology, Medicine / Leiden University Medical Center (LUMC), Leiden University, Leiden
hdl
1887/72412
ISBN(s)
9789463324892