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 …

[HTML][HTML] Research models and gene augmentation therapy for CRB1 retinal dystrophies

N Boon, J Wijnholds, LP Pellissier - Frontiers in Neuroscience, 2020 - frontiersin.org
Retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) are inherited degenerative
retinal dystrophies with vision loss that ultimately lead to blindness. Several genes have …

[HTML][HTML] Retinal dystrophies and the road to treatment: clinical requirements and considerations

M Talib, CJF Boon - Asia-Pacific Journal of Ophthalmology, 2020 - Elsevier
Retinal dystrophies (RDs) comprise relatively rare but devastating causes of progressive
vision loss. They represent a spectrum of diseases with marked genetic and clinical …

CRB2 loss in rod photoreceptors is associated with progressive loss of retinal contrast sensitivity

CH Alves, N Boon, AA Mulder, AJ Koster… - International Journal of …, 2019 - mdpi.com
Variations in the Crumbs homolog-1 (CRB1) gene are associated with a wide variety of
autosomal recessive retinal dystrophies, including early onset retinitis pigmentosa (RP) and …

Defining inclusion criteria and endpoints for clinical trials: a prospective cross‐sectional study in CRB1‐associated retinal dystrophies

M Talib, MJ van Schooneveld, J Wijnholds… - Acta …, 2021 - Wiley Online Library
Purpose To investigate the retinal structure and function in patients with CRB1‐associated
retinal dystrophies (RD) and to explore potential clinical endpoints. Methods In this …

Human CRB1 and CRB2 form homo-and heteromeric protein complexes in the retina

IF Stehle, JA Imventarza, F Woerz… - Life Science …, 2024 - life-science-alliance.org
Crumbs homolog 1 (CRB1) is one of the key genes linked to retinitis pigmentosa and Leber
congenital amaurosis, which are characterized by a high clinical heterogeneity. The Crumbs …

Recombinant AAV-crumbs homologue composition and methods for treating LCA-8 and progressive RP

J Wijnholds, LPF Pellissier - US Patent 11,246,947, 2022 - Google Patents
The present invention relates to a Crumbs homologue (CRB) therapeutic for use as a
medicament or in a method of treatment or prophylaxis, for example in the treatment or …

Clinical and Therapeutic Evaluation of the Ten Most Prevalent CRB1 Mutations

B Lopes da Costa, M Kolesnikova, SR Levi, T Cabral… - Biomedicines, 2023 - mdpi.com
Mutations in the Crumbs homolog 1 (CRB1) gene lead to severe inherited retinal
dystrophies (IRDs), accounting for nearly 80,000 cases worldwide. To date, there is no …

Defining phenotype, tropism, and retinal gene therapy using adeno-associated viral vectors (AAVs) in new-born Brown Norway rats with a spontaneous mutation in …

N Boon, CH Alves, AA Mulder, CA Andriessen… - International Journal of …, 2021 - mdpi.com
Mutations in the Crumbs homologue 1 (CRB1) gene cause inherited retinal dystrophies,
such as early-onset retinitis pigmentosa and Leber congenital amaurosis. A Brown Norway …

Diverse functions and pathogenetic role of Crumbs in retinopathy

X Zhou, L Zhao, C Wang, W Sun, B Jia, D Li… - Cell Communication and …, 2024 - Springer
The Crumbs protein (CRB) family plays a crucial role in maintaining the apical–basal
polarity and integrity of embryonic epithelia. The family comprises different isoforms in …