[PDF][PDF] AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype
N Boon, X Lu, CA Andriessen, I Moustakas, TM Buck… - Stem cell reports, 2023 - cell.com
Retinitis pigmentosa and Leber congenital amaurosis are inherited retinal dystrophies that
can be caused by mutations in the Crumbs homolog 1 (CRB1) gene. CRB1 is required for …
can be caused by mutations in the Crumbs homolog 1 (CRB1) gene. CRB1 is required for …
Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models
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 …
retinal dystrophies. Gene transfer therapy is the most promising cure for retinal dystrophies …
[HTML][HTML] AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model
TM Buck, RM Vos, CH Alves, J Wijnholds - Molecular Therapy-Methods & …, 2021 - cell.com
Loss of Crumbs homolog 1 (CRB1) or CRB2 proteins in Müller cells or photoreceptors in the
mouse retina results in a CRB dose-dependent retinal phenotype. In this study, we present a …
mouse retina results in a CRB dose-dependent retinal phenotype. In this study, we present a …
[HTML][HTML] Specific tools for targeting and expression in Müller glial cells
LP Pellissier, RM Hoek, RM Vos, WM Aartsen… - … Therapy-Methods & …, 2014 - cell.com
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 …
disease pathogenesis and are an interesting target for therapeutic approaches for retinal …
[PDF][PDF] Gene therapy of dominant CRX-Leber congenital amaurosis using patient stem cell-derived retinal organoids
Mutations in the photoreceptor transcription factor gene cone-rod homeobox (CRX) lead to
distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber …
distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber …
Targeted ablation of Crb2 in photoreceptor cells induces retinitis pigmentosa
CH Alves, LP Pellissier, RM Vos… - Human molecular …, 2014 - academic.oup.com
In humans, the Crumbs homolog-1 (CRB1) gene is mutated in autosomal recessive Leber
congenital amaurosis and early-onset retinitis pigmentosa. In mammals, the Crumbs family …
congenital amaurosis and early-onset retinitis pigmentosa. In mammals, the Crumbs family …
[PDF][PDF] Human iPSC-derived retinas recapitulate the fetal CRB1 CRB2 complex formation and demonstrate that photoreceptors and Müller glia are targets of AAV5
Human retinal organoids from induced pluripotent stem cells (hiPSCs) can be used to
confirm the localization of proteins in retinal cell types and to test transduction and …
confirm the localization of proteins in retinal cell types and to test transduction and …
Loss of CRB2 in Müller glial cells modifies a CRB1-associated retinitis pigmentosa phenotype into a Leber congenital amaurosis phenotype
PM Quinn, AA Mulder, C Henrique Alves… - Human Molecular …, 2019 - academic.oup.com
Variations in the human Crumbs homolog-1 (CRB1) gene lead to an array of retinal
dystrophies including early onset of retinitis pigmentosa (RP) and Leber congenital …
dystrophies including early onset of retinitis pigmentosa (RP) and Leber congenital …
[HTML][HTML] Targeted Ablation of Crb1 and Crb2 in Retinal Progenitor Cells Mimics Leber Congenital Amaurosis
Development in the central nervous system is highly dependent on the regulation of the
switch from progenitor cell proliferation to differentiation, but the molecular and cellular …
switch from progenitor cell proliferation to differentiation, but the molecular and cellular …
A single amino acid substitution (Cys249Trp) in Crb1 causes retinal degeneration and deregulates expression of pituitary tumor transforming gene Pttg1
SA van de Pavert, J Meuleman… - Journal of …, 2007 - Soc Neuroscience
Different mutations in the human Crumbs homolog-1 (CRB1) gene cause a variety of retinal
dystrophies, such as Leber congenital amaurosis, early onset retinitis pigmentosa (eg …
dystrophies, such as Leber congenital amaurosis, early onset retinitis pigmentosa (eg …