Advancing cell therapy for neurodegenerative diseases

S Temple - Cell stem cell, 2023 - cell.com
Cell-based therapies are being developed for various neurodegenerative diseases that
affect the central nervous system (CNS). Concomitantly, the roles of individual cell types in …

Integrating population genetics, stem cell biology and cellular genomics to study complex human diseases

N Farbehi, DR Neavin, ASE Cuomo, L Studer… - Nature Genetics, 2024 - nature.com
Human pluripotent stem (hPS) cells can, in theory, be differentiated into any cell type,
making them a powerful in vitro model for human biology. Recent technological advances …

Exploring the contribution of ARMS2 and HTRA1 genetic risk factors in age-related macular degeneration

Y Pan, Y Fu, PN Baird, RH Guymer, T Das… - Progress in Retinal and …, 2023 - Elsevier
Age-related macular degeneration (AMD) is the leading cause of severe irreversible central
vision loss in individuals over 65 years old. Genome-wide association studies (GWASs) …

A systems biology approach uncovers novel disease mechanisms in age-related macular degeneration

LD Orozco, LA Owen, J Hofmann, AD Stockwell, J Tao… - Cell genomics, 2023 - cell.com
Age-related macular degeneration (AMD) is a leading cause of blindness, affecting 200
million people worldwide. To identify genes that could be targeted for treatment, we created …

Human iPSC-based disease modeling studies identify a common mechanistic defect and potential therapies for AMD and related macular dystrophies

S Dalvi, M Roll, A Chatterjee, LK Kumar, A Bhogavalli… - Developmental Cell, 2024 - cell.com
Age-related macular degeneration (AMD) and related macular dystrophies (MDs) primarily
affect the retinal pigment epithelium (RPE) in the eye. A hallmark of AMD/MDs that drives …

Single-Cell RNA sequencing in Dissecting Microenvironment of Age-related Macular Degeneration: Challenges and Perspectives

Y Tan, J Huang, D Li, C Zou, D Liu, B Qin - Ageing Research Reviews, 2023 - Elsevier
Age-related macular degeneration (AMD) is the leading cause of blindness in individuals
over the age of 50 years, yet its etiology and pathogenesis largely remain uncovered. Single …

[HTML][HTML] Genome-wide meta-analysis identifies risk loci and improves disease prediction of age-related macular degeneration

W He, X Han, JS Ong, Y Wu, AW Hewitt, DA Mackey… - Ophthalmology, 2024 - Elsevier
Purpose To identify age-related macular degeneration (AMD) risk loci and to establish a
polygenic prediction model. Design Genome-wide association study (GWAS) and polygenic …

[HTML][HTML] Pluripotent stem cell-derived models of retinal disease: Elucidating pathogenesis, evaluating novel treatments, and estimating toxicity

M Kurzawa-Akanbi, N Tzoumas… - Progress in Retinal and …, 2024 - Elsevier
Blindness poses a growing global challenge, with approximately 26% of cases attributed to
degenerative retinal diseases. While gene therapy, optogenetic tools, photosensitive …

Dissecting Regulators of Aging and Age‐Related Macular Degeneration in the Retinal Pigment Epithelium

PP Karunadharma, RJ Kapphahn… - Oxidative Medicine …, 2022 - Wiley Online Library
Age‐related macular degeneration (AMD), the leading cause of blindness in elderly
populations, involves the loss of central vision due to progressive dysfunction of the retinal …

Transcriptomic and chromatin accessibility analysis of the human macular and peripheral retinal pigment epithelium at the single-cell level

NK Mullin, AP Voigt, EA Boese, X Liu, EM Stone… - The American journal of …, 2023 - Elsevier
Some human retinal diseases are characterized by pathology that is restricted to specific cell
types and to specific regions of the eye. Several disease entities either selectively affect or …