Pluripotent stem cells in disease modelling and drug discovery

Y Avior, I Sagi, N Benvenisty - Nature reviews Molecular cell biology, 2016 - nature.com
Experimental modelling of human disorders enables the definition of the cellular and
molecular mechanisms underlying diseases and the development of therapies for treating …

Human-induced pluripotent stem cells: potential for neurodegenerative diseases

CA Ross, SS Akimov - Human molecular genetics, 2014 - academic.oup.com
The cell biology of human neurodegenerative diseases has been difficult to study till
recently. The development of human induced pluripotent stem cell (iPSC) models has …

From confluent human iPS cells to self-forming neural retina and retinal pigmented epithelium

S Reichman, A Terray, A Slembrouck… - Proceedings of the …, 2014 - National Acad Sciences
Progress in retinal-cell therapy derived from human pluripotent stem cells currently faces
technical challenges that require the development of easy and standardized protocols. Here …

[HTML][HTML] Sialylated IgG induces the transcription factor REST in alveolar macrophages to protect against lung inflammation and severe influenza disease

S Chakraborty, BYL Cheng, DL Edwards, JC Gonzalez… - Immunity, 2025 - cell.com
While most respiratory viral infections resolve with little harm to the host, severe symptoms
arise when infection triggers an aberrant inflammatory response that damages lung tissue …

A conserved YAP/Notch/REST network controls the neuroendocrine cell fate in the lungs

YT Shue, AP Drainas, NY Li, SM Pearsall… - Nature …, 2022 - nature.com
The Notch pathway is a conserved cell-cell communication pathway that controls cell fate
decisions. Here we sought to determine how Notch pathway activation inhibits the …

Multiscale modeling uncovers 7q11. 23 copy number variation–dependent changes in ribosomal biogenesis and neuronal maturation and excitability

M Mihailovich, PL Germain, R Shyti, D Pozzi… - The Journal of clinical …, 2024 - jci.org
Copy number variation (CNV) at 7q11. 23 causes Williams-Beuren syndrome (WBS) and 7q
microduplication syndrome (7Dup), neurodevelopmental disorders (NDDs) featuring …

Large-scale generation of human iPSC-derived neural stem cells/early neural progenitor cells and their neuronal differentiation

L D'Aiuto, Y Zhi, D Kumar Das, MR Wilcox… - …, 2014 - Taylor & Francis
Induced pluripotent stem cell (iPSC)-based technologies offer an unprecedented opportunity
to perform high-throughput screening of novel drugs for neurological and …

Huntington disease as a neurodevelopmental disorder and early signs of the disease in stem cells

K Wiatr, WJ Szlachcic, M Trzeciak, M Figlerowicz… - Molecular …, 2018 - Springer
Huntington disease (HD) is a dominantly inherited disorder caused by a CAG expansion
mutation in the huntingtin (HTT) gene, which results in the HTT protein that contains an …

Transcription, epigenetics and ameliorative strategies in Huntington's Disease: a genome-wide perspective

LM Valor - Molecular neurobiology, 2015 - Springer
Transcriptional dysregulation in Huntington's disease (HD) is an early event that shapes the
brain transcriptome by both the depletion and ectopic activation of gene products that …

Prototypical oncogene family Myc defines unappreciated distinct lineage states of small cell lung cancer

AS Patel, S Yoo, R Kong, T Sato, A Sinha, S Karam… - Science …, 2021 - science.org
Comprehensive genomic analyses of small cell lung cancer (SCLC) have revealed frequent
mutually exclusive genomic amplification of MYC family members. Hence, it has been long …