The lncRNA H19 alleviates muscular dystrophy by stabilizing dystrophin

Y Zhang, Y Li, Q Hu, Y Xi, Z Xing, Z Zhang, L Huang… - Nature cell …, 2020 - nature.com
Dystrophin proteomic regulation in muscular dystrophies (MDs) remains unclear. We report
that a long noncoding RNA (lncRNA), H19, associates with dystrophin and inhibits E3-ligase …

A concise review on induced pluripotent stem cell-derived cardiomyocytes for personalized regenerative medicine

P Pushp, DES Nogueira, CAV Rodrigues… - Stem cell reviews and …, 2021 - Springer
The induced pluripotent stem cells (iPSCs) are derived from somatic cells by using
reprogramming factors such as Oct4, Sox2, Klf4, and c-Myc (OSKM) or Oct4, Sox2, Nanog …

Can microfluidics address biomanufacturing challenges in drug/gene/cell therapies?

HF Chan, S Ma, KW Leong - Regenerative biomaterials, 2016 - academic.oup.com
Translation of any inventions into products requires manufacturing. Development of
drug/gene/cell delivery systems will eventually face manufacturing challenges, which …

Generation of human iPSCs from cells of fibroblastic and epithelial origin by means of the oriP/EBNA-1 episomal reprogramming system

AM Drozd, MP Walczak, S Piaskowski… - Stem cell research & …, 2015 - Springer
Introduction The prospect of therapeutic applications of the induced pluripotent stem cells
(iPSCs) is based on their ability to generate virtually any cell type present in human body …

Patient-specific iPSC-derived endothelial cells provide long-term phenotypic correction of hemophilia A

C Olgasi, M Talmon, S Merlin, A Cucci… - Stem cell reports, 2018 - cell.com
We generated patient-specific disease-free induced pluripotent stem cells (iPSCs) from
peripheral blood CD34+ cells and differentiated them into functional endothelial cells (ECs) …

CRISPR‐Cas9‐based genome editing of human induced pluripotent stem cells

JC Giacalone, TP Sharma, ER Burnight… - Current protocols in …, 2018 - Wiley Online Library
Human induced pluripotent stem cells (hiPSCs) are the ideal cell source for autologous cell
replacement. However, for patients with Mendelian diseases, genetic correction of the …

Tapping stem cells to target AMD: challenges and prospects

C Brandl, F Grassmann, J Riolfi, BHF Weber - Journal of clinical medicine, 2015 - mdpi.com
Human pluripotent stem cells (hPSCs) are increasingly gaining attention in biomedicine as
valuable resources to establish patient-derived cell culture models of the cell type known to …

Direct control of stem cell behavior using biomaterials and genetic factors

JK Yoon, ML Kang, JH Park, KM Lee… - Stem Cells …, 2018 - Wiley Online Library
Stem cells have recently emerged as an important candidate for cell therapy. However,
some major limitations still exist such as a small quantity of cell supply, senescence, and …

Bioreactor model of neuromuscular junction with electrical stimulation for pharmacological potency testing

SN Charoensook, DJ Williams, S Chakraborty… - Integrative …, 2017 - academic.oup.com
In vitro models of the neuromuscular junction (NMJ) are emerging as a valuable tool to study
synaptogenesis, synaptic maintenance, and pathogenesis of neurodegenerative diseases …

Tools for Targeted Genome Engineering of Established Drosophila Cell Lines

L Cherbas, J Hackney, L Gong, C Salzer, E Mauser… - Genetics, 2015 - academic.oup.com
We describe an adaptation of φC31 integrase–mediated targeted cassette exchange for use
in Drosophila cell lines. Single copies of an attP-bounded docking platform carrying a GFP …