[PDF][PDF] The library of integrated network-based cellular signatures NIH program: system-level cataloging of human cells response to perturbations

…, D Sareen, L Ornelas, M Banuelos, B Mandefro… - Cell systems, 2018 - cell.com
The Library of Integrated Network-Based Cellular Signatures (LINCS) is an NIH Common
Fund program that catalogs how human cells globally respond to chemical, genetic, and …

[HTML][HTML] Aneuploidy induces profound changes in gene expression, proliferation and tumorigenicity of human pluripotent stem cells

U Ben-David, G Arad, U Weissbein, B Mandefro… - Nature …, 2014 - nature.com
Human pluripotent stem cells (hPSCs) tend to acquire genomic aberrations in culture, the
most common of which is trisomy of chromosome 12. Here we dissect the cellular and …

[PDF][PDF] Human iPSC-derived endothelial cells and microengineered organ-chip enhance neuronal development

…, A Laperle, A Meyer, M Godoy, PS Kay, B Mandefro… - Stem cell reports, 2018 - cell.com
… (B) Single population histogram of Chips seeded only with non-GFP BMECs (black) or
GFP-spMNs (green) that defined negative and positive fractions, respectively. Number of events is …

HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity

…, A Sahabian, L Lenaeus, B Mandefro… - Human molecular …, 2015 - academic.oup.com
Huntington's disease (HD) is a fatal neurodegenerative disease, caused by expansion of
polyglutamine repeats in the Huntingtin gene, with longer expansions leading to earlier ages of …

Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy

…, HE Johansson, B Mandefro… - Proceedings of the …, 2017 - National Acad Sciences
Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by progressive
motor neuron loss and caused by mutations in SMN1 (Survival Motor Neuron 1). The …

Human embryonic stem cells as models for aneuploid chromosomal syndromes

JC Biancotti, K Narwani, N Buehler, B Mandefro… - Stem …, 2010 - academic.oup.com
Syndromes caused by chromosomal aneuploidies are widely recognized genetic disorders
in humans and often lead to spontaneous miscarriage. Preimplantation genetic screening is …

Reliable generation of induced pluripotent stem cells from human lymphoblastoid cell lines

…, L Ornelas, N Yeager, B Mandefro… - Stem cells …, 2014 - academic.oup.com
… These lines are established by infecting resting peripheral B lymphocytes isolated from
simple blood draws with Epstein Barr virus (EBV) to give rise to actively proliferating LCLs [9]. …

[HTML][HTML] Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines

…, E Galvez, D Perez, I Meepe, S Lei, B Mandefro… - Nature …, 2022 - nature.com
Answer ALS is a biological and clinical resource of patient-derived, induced pluripotent
stem (iPS) cell lines, multi-omic data derived from iPS neurons and longitudinal clinical and …

[HTML][HTML] Spinal muscular atrophy patient iPSC-derived motor neurons have reduced expression of proteins important in neuronal development

HR Fuller, B Mandefro, SL Shirran, AR Gross… - Frontiers in cellular …, 2016 - frontiersin.org
Spinal muscular atrophy (SMA) is an inherited neuromuscular disease primarily characterized
by degeneration of spinal motor neurons, and caused by reduced levels of the SMN …

[PDF][PDF] An integrated multi-omic analysis of iPSC-derived motor neurons from C9ORF72 ALS patients

…, R Holewenski, S Lei, A Lenail, L Lima, B Mandefro… - Iscience, 2021 - cell.com
Neurodegenerative diseases are challenging for systems biology because of the lack of
reliable animal models or patient samples at early disease stages. Induced pluripotent stem …