Screening out irrelevant cell-based models of disease

P Horvath, N Aulner, M Bickle, AM Davies… - Nature reviews Drug …, 2016 - nature.com
The common and persistent failures to translate promising preclinical drug candidates into
clinical success highlight the limited effectiveness of disease models currently used in drug …

Alveolar macrophages in tissue homeostasis, inflammation, and infection: evolving concepts of therapeutic targeting

C Malainou, SM Abdin, N Lachmann… - The Journal of …, 2023 - Am Soc Clin Investig
Alveolar macrophages (AMs) are the sentinel cells of the alveolar space, maintaining
homeostasis, fending off pathogens, and controlling lung inflammation. During acute lung …

Bestrophin 1 and retinal disease

AA Johnson, KE Guziewicz, CJ Lee… - Progress in retinal and …, 2017 - Elsevier
Mutations in the gene BEST1 are causally associated with as many as five clinically distinct
retinal degenerative diseases, which are collectively referred to as the “bestrophinopathies” …

Pluripotent stem cell-derived cerebral organoids reveal human oligodendrogenesis with dorsal and ventral origins

H Kim, R Xu, R Padmashri, A Dunaevsky, Y Liu… - Stem cell reports, 2019 - cell.com
The process of oligodendrogenesis has been relatively well delineated in the rodent brain.
However, it remains unknown whether analogous developmental processes are manifested …

Good cell culture practice for stem cells and stem-cell-derived models

D Pamies, A Bal-Price, A Simeonov, D Tagle, D Allen… - 2017 - kops.uni-konstanz.de
The first guidance on Good Cell Culture Practice dates back to 2005. This document
expands this to aspects of quality assurance for in vitro cell culture focusing on the …

[HTML][HTML] Efficient in vivo liver-directed gene editing using CRISPR/Cas9

K Singh, H Evens, N Nair, MY Rincón, S Sarcar… - Molecular Therapy, 2018 - cell.com
In vivo tissue-specific genome editing at the desired loci is still a challenge. Here, we report
that AAV9-delivery of truncated guide RNAs (gRNAs) and Cas9 under the control of a …

[HTML][HTML] Clinical aspects of neurointestinal disease: Pathophysiology, diagnosis, and treatment

AM Goldstein, N Thapar, TB Karunaratne… - Developmental …, 2016 - Elsevier
The enteric nervous system (ENS) is involved in the regulation of virtually all gut functions.
Conditions referred to as enteric neuropathies are the result of various mechanisms …

Generation and application of novel hES cell reporter lines for the differentiation and maturation of hPS cell-derived islet-like clusters

E Zanfrini, M Bandral, L Jarc, MA Ramirez-Torres… - Scientific Reports, 2024 - nature.com
The significant advances in the differentiation of human pluripotent stem (hPS) cells into
pancreatic endocrine cells, including functional β-cells, have been based on a detailed …

Concise review: making and using clinically compliant pluripotent stem cell lines

MK Carpenter, MS Rao - Stem cells translational medicine, 2015 - academic.oup.com
The field of pluripotent stem cells (PSCs) is in a state of dynamic flux driven by significant
advances in the derivation of specific phenotypes from embryonic stem cells, breakthroughs …

Fish genome manipulation and directional breeding

D Ye, ZY Zhu, YH Sun - Science China Life Sciences, 2015 - Springer
Aquaculture is one of the fastest develo** agricultural industries worldwide. One of the
most important factors for sustainable aquaculture is the development of high performing …