Current challenges of iPSC-based disease modeling and therapeutic implications

MX Doss, A Sachinidis - Cells, 2019 - mdpi.com
Induced pluripotent stem cell (iPSC)-based disease modelling and the cell replacement
therapy approach have proven to be very powerful and instrumental in biomedical research …

Induced pluripotent stem cells (iPSCs)—roles in regenerative therapies, disease modelling and drug screening

MAM Aboul-Soud, AJ Alzahrani, A Mahmoud - Cells, 2021 - mdpi.com
The discovery of induced pluripotent stem cells (iPSCs) has made an invaluable contribution
to the field of regenerative medicine, paving way for identifying the true potential of human …

Adenine base editor engineering reduces editing of bystander cytosines

YK Jeong, SH Lee, GH Hwang, SA Hong, S Park… - Nature …, 2021 - nature.com
Adenine base editors (ABEs) catalyze specific A-to-G conversions at genomic sites of
interest. However, ABEs also induce cytosine deamination at the target site. To reduce the …

The RASopathy family: consequences of germline activation of the RAS/MAPK pathway

M Tajan, R Paccoud, S Branka, T Edouard… - Endocrine …, 2018 - academic.oup.com
Abstract Noonan syndrome [NS; Mendelian Inheritance in Men (MIM)# 163950] and related
syndromes [Noonan syndrome with multiple lentigines (formerly called LEOPARD …

Juvenile myelomonocytic leukemia: who's the driver at the wheel?

CM Niemeyer, C Flotho - Blood, The Journal of the American …, 2019 - ashpublications.org
Juvenile myelomonocytic leukemia (JMML) is a unique clonal hematopoietic disorder of
early childhood. It is classified as an overlap myeloproliferative/myelodysplastic neoplasm …

Osteosarcoma: molecular pathogenesis and iPSC modeling

YH Lin, BE Jewell, J Gingold, L Lu, R Zhao… - Trends in molecular …, 2017 - cell.com
Rare hereditary disorders provide unequivocal evidence of the importance of genes in
human disease pathogenesis. Familial syndromes that predispose to osteosarcomagenesis …

Patient-derived induced pluripotent stem cells in cancer research and precision oncology

EP Papapetrou - Nature medicine, 2016 - nature.com
Together with recent advances in the processing and culture of human tissue,
bioengineering, xenotransplantation and genome editing, Induced pluripotent stem cells …

6-Amino-3-methylpyrimidinones as potent, selective, and orally efficacious SHP2 inhibitors

P Sarver, M Acker, JT Bagdanoff, Z Chen… - Journal of medicinal …, 2019 - ACS Publications
Protein tyrosine phosphatase SHP2 is an oncoprotein associated with cancer as well as a
potential immune modulator because of its role in the programmed cell death PD-L1/PD-1 …

Turning the tide in myelodysplastic/myeloproliferative neoplasms

MWN Deininger, JW Tyner, E Solary - Nature Reviews Cancer, 2017 - nature.com
Myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) are aggressive
myeloid malignancies recognized as a distinct category owing to their unique combination of …

The role of GATA2 in adult hematopoiesis and cell fate determination

IJA Peters, E de Pater, W Zhang - Frontiers in Cell and …, 2023 - frontiersin.org
The correct maintenance and differentiation of hematopoietic stem cells (HSC) in bone
marrow is vital for the maintenance and operation of the human blood system. GATA2 plays …