Induced pluripotent stem cells and their use in human models of disease and development

P Karagiannis, K Takahashi, M Saito… - Physiological …, 2019 - journals.physiology.org
The discovery of somatic cell nuclear transfer proved that somatic cells can carry the same
genetic code as the zygote, and that activating parts of this code are sufficient to reprogram …

Investigating human disease using stem cell models

JL Sterneckert, P Reinhardt, HR Schöler - Nature Reviews Genetics, 2014 - nature.com
Tractable and accurate disease models are essential for understanding disease
pathogenesis and for developing new therapeutics. As stem cells are capable of self …

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

P Xu, Y Tong, X Liu, T Wang, L Cheng, B Wang, X Lv… - Scientific reports, 2015 - nature.com
Abstract β-Thalassemia is one of the most common genetic blood diseases and is caused by
either point mutations or deletions in the β-globin (HBB) gene. The generation of patient …

Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system

B Song, Y Fan, W He, D Zhu, X Niu… - Stem cells and …, 2015 - liebertpub.com
The generation of beta-thalassemia (β-Thal) patient-specific induced pluripotent stem cells
(iPSCs), subsequent homologous recombination-based gene correction of disease-causing …

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

A Filareto, S Parker, R Darabi, L Borges… - Nature …, 2013 - nature.com
Duchenne muscular dystrophy is a progressive and incurable neuromuscular disease
caused by genetic and biochemical defects of the dystrophin–glycoprotein complex. Here …

Effects of genetic correction on the differentiation of hair cell-like cells from iPSCs with MYO15A mutation

JR Chen, ZH Tang, J Zheng, HS Shi, J Ding… - Cell Death & …, 2016 - nature.com
Deafness or hearing loss is a major issue in human health. Inner ear hair cells are the main
sensory receptors responsible for hearing. Defects in hair cells are one of the major causes …

Combining single strand oligodeoxynucleotides and CRISPR/Cas9 to correct gene mutations in β-thalassemia-induced pluripotent stem cells

X Niu, W He, B Song, Z Ou, D Fan, Y Chen… - Journal of Biological …, 2016 - ASBMB
β-Thalassemia (β-Thal) is one of the most common genetic diseases in the world. The
generation of patient-specific β-Thal-induced pluripotent stem cells (iPSCs), correction of the …

hi PSC‐derived iMSC s: NextGen MSC s as an advanced therapeutically active cell resource for regenerative medicine

V Sabapathy, S Kumar - Journal of cellular and molecular …, 2016 - Wiley Online Library
Mesenchymal stem cells (MSC s) are being assessed for ameliorating the severity of graft‐
versus‐host disease, autoimmune conditions, musculoskeletal injuries and cardiovascular …

Generation of tooth-like structures from integration-free human urine induced pluripotent stem cells

J Cai, Y Zhang, P Liu, S Chen, X Wu, Y Sun, A Li… - Cell regeneration, 2013 - Springer
Background Tooth is vital not only for a good smile, but also good health. Yet, we lose tooth
regularly due to accidents or diseases. An ideal solution to this problem is to regenerate …

The combination of CRISPR/Cas9 and iPSC technologies in the gene therapy of human β-thalassemia in mice

Z Ou, X Niu, W He, Y Chen, B Song, Y Xian, D Fan… - Scientific reports, 2016 - nature.com
Abstract β-thalassemia results from point mutations or small deletions in the β-globin (HBB)
gene that ultimately cause anemia. The generation of induced pluripotent stem cells (iPSCs) …