[HTML][HTML] Recombinant adeno-associated viral vectors (rAAV)-vector elements in ocular gene therapy clinical trials and transgene expression and bioactivity assays
TM Buck, J Wijnholds - International journal of molecular sciences, 2020 - mdpi.com
Inherited retinal dystrophies and optic neuropathies cause chronic disabling loss of visual
function. The development of recombinant adeno-associated viral vectors (rAAV) gene …
function. The development of recombinant adeno-associated viral vectors (rAAV) gene …
[HTML][HTML] Organoids and organ chips in ophthalmology
Recent advances have driven the development of stem cell-derived, self-organizing, three-
dimensional miniature organs, termed organoids, which mimic different eye tissues …
dimensional miniature organs, termed organoids, which mimic different eye tissues …
[PDF][PDF] Human brain organoids assemble functionally integrated bilateral optic vesicles
E Gabriel, W Albanna, G Pasquini, A Ramani… - Cell Stem Cell, 2021 - cell.com
During embryogenesis, optic vesicles develop from the diencephalon via a multistep
process of organogenesis. Using induced pluripotent stem cell (iPSC)-derived human brain …
process of organogenesis. Using induced pluripotent stem cell (iPSC)-derived human brain …
[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 …
[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 …
[PDF][PDF] CRB1 is required for recycling by RAB11A+ vesicles in human retinal organoids
TM Buck, PMJ Quinn, LP Pellissier, AA Mulder… - Stem cell reports, 2023 - cell.com
CRB1 gene mutations can cause early-or late-onset retinitis pigmentosa, Leber congenital
amaurosis, or maculopathy. Recapitulating human CRB1 phenotypes in animal models has …
amaurosis, or maculopathy. Recapitulating human CRB1 phenotypes in animal models has …
Affinity-controlled release of rod-derived cone viability factor enhances cone photoreceptor survival
Retinitis pigmentosa (RP) is a group of genetic diseases that results in rod photoreceptor
cell degeneration, which subsequently leads to cone photoreceptor cell death, impaired …
cell degeneration, which subsequently leads to cone photoreceptor cell death, impaired …
[HTML][HTML] Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/− retinal organoids
N Boon, X Lu, CA Andriessen, M Orlovà… - … Therapy-Methods & …, 2023 - cell.com
The majority of patients with mutations in CRB1 develop either early-onset retinitis
pigmentosa as young children or Leber congenital amaurosis as newborns. The cause for …
pigmentosa as young children or Leber congenital amaurosis as newborns. The cause for …
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] Retinogenesis of the human fetal retina: an apical polarity perspective
PMJ Quinn, J Wijnholds - Genes, 2019 - mdpi.com
The Crumbs complex has prominent roles in the control of apical cell polarity, in the coupling
of cell density sensing to downstream cell signaling pathways, and in regulating junctional …
of cell density sensing to downstream cell signaling pathways, and in regulating junctional …