Human disease models in drug development

A Loewa, JJ Feng, S Hedtrich - Nature reviews bioengineering, 2023 - nature.com
Biomedical research is undergoing a paradigm shift towards approaches centred on human
disease models owing to the notoriously high failure rates of the current drug development …

Retinal organoids: a window into human retinal development

M O'Hara-Wright, A Gonzalez-Cordero - Development, 2020 - journals.biologists.com
Retinal development and maturation are orchestrated by a series of interacting signalling
networks that drive the morphogenetic transformation of the anterior developing brain …

A look into retinal organoids: methods, analytical techniques, and applications

TAV Afanasyeva, JC Corral-Serrano, A Garanto… - Cellular and Molecular …, 2021 - Springer
Inherited retinal diseases (IRDs) cause progressive loss of light-sensitive photoreceptors in
the eye and can lead to blindness. Gene-based therapies for IRDs have shown remarkable …

Suspension culture improves iPSC expansion and pluripotency phenotype

N Cuesta-Gomez, K Verhoeff, N Dadheech… - Stem Cell Research & …, 2023 - Springer
Background Induced pluripotent stem cells (iPSCs) offer potential to revolutionize
regenerative medicine as a renewable source for islets, dopaminergic neurons, retinal cells …

Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: Applications, challenges, and emerging opportunities

Y Chien, YJ Hsiao, SJ Chou, TY Lin… - Journal of …, 2022 - Springer
Abstract Inherited Retinal Diseases (IRDs) are considered one of the leading causes of
blindness worldwide. However, the majority of them still lack a safe and effective treatment …

Emerging strategies of engineering retinal organoids and organoid-on-a-chip in modeling intraocular drug delivery: Current progress and future perspectives

J Yu, Y Yin, Y Leng, J Zhang, C Wang, Y Chen… - Advanced Drug Delivery …, 2023 - Elsevier
Retinal diseases are a rising concern as major causes of blindness in an aging society;
therapeutic options are limited, and the precise pathogenesis of these diseases remains …

[HTML][HTML] Human neural organoids: Models for developmental neurobiology and disease

B Guy, JS Zhang, LH Duncan, RJ Johnston Jr - Developmental biology, 2021 - Elsevier
Human organoids stand at the forefront of basic and translational research, providing
experimentally tractable systems to study human development and disease. These stem cell …

[HTML][HTML] Tropism of AAV vectors in photoreceptor-like cells of human iPSC-derived retinal organoids

ME McClements, H Steward, W Atkin… - … Vision Science & …, 2022 - iovs.arvojournals.org
Purpose: To expand the use of human retinal organoids from induced pluripotent stem cells
(iPSCs) as an in vitro model of the retina for assessing gene therapy treatments, it is …

Retinal organoids and retinal prostheses: an overview

A Bellapianta, A Cetkovic, M Bolz, A Salti - International journal of …, 2022 - mdpi.com
Despite the progress of modern medicine in the last decades, millions of people diagnosed
with retinal dystrophies (RDs), such as retinitis pigmentosa, or age-related diseases, such as …

[HTML][HTML] A simple and efficient method for generating human retinal organoids

F Regent, HY Chen, RA Kelley, Z Qu, A Swaroop… - Molecular …, 2020 - ncbi.nlm.nih.gov
Purpose Retinal organoids (ROs) derived from human pluripotent stem cells largely
recapitulate key features of in vivo retinal development, thus permitting the study of …