Treg cell-based therapies: challenges and perspectives

C Raffin, LT Vo, JA Bluestone - Nature Reviews Immunology, 2020 - nature.com
Cellular therapies using regulatory T (Treg) cells are currently undergoing clinical trials for
the treatment of autoimmune diseases, transplant rejection and graft-versus-host disease. In …

Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease

MD Hoban, SH Orkin, DE Bauer - Blood, The Journal of the …, 2016 - ashpublications.org
Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent
and severe monogenic disorder, SCD has been long considered a logical candidate for …

Generation of transgene-free hematopoietic stem cells from human induced pluripotent stem cells

O Piau, M Brunet-Manquat, B L'homme, L Petit… - Cell Stem Cell, 2023 - cell.com
Hematopoietic stem cells (HSCs) are the rare cells responsible for the lifelong curative
effects of hematopoietic cell (HC) transplantation. The demand for clinical-grade HSCs has …

[HTML][HTML] Embryonic endothelial evolution towards first hematopoietic stem cells revealed by single-cell transcriptomic and functional analyses

S Hou, Z Li, X Zheng, Y Gao, J Dong, Y Ni, X Wang… - Cell research, 2020 - nature.com
Hematopoietic stem cells (HSCs) in adults are believed to be born from hemogenic
endothelial cells (HECs) in mid-gestational embryos. Due to the rare and transient nature …

[HTML][HTML] Fetal liver hematopoiesis: from development to delivery

K Lewis, M Yoshimoto, T Takebe - Stem Cell Research & Therapy, 2021 - Springer
Clinical transplants of hematopoietic stem cells (HSC) can provide a lifesaving therapy for
many hematological diseases; however, therapeutic applications are hampered by donor …

[HTML][HTML] Stage-specific human induced pluripotent stem cells map the progression of myeloid transformation to transplantable leukemia

AG Kotini, CJ Chang, A Chow, H Yuan, TC Ho, T Wang… - Cell stem cell, 2017 - cell.com
Myeloid malignancy is increasingly viewed as a disease spectrum, comprising
hematopoietic disorders that extend across a phenotypic continuum ranging from clonal …

A view of human haematopoietic development from the Petri dish

A Ditadi, CM Sturgeon, G Keller - Nature Reviews Molecular Cell …, 2017 - nature.com
Human pluripotent stem cells (hPSCs) provide an unparalleled opportunity to establish in
vitro differentiation models that will transform our approach to the study of human …

Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors

S Doulatov, LT Vo, ER Macari, L Wahlster… - Science translational …, 2017 - science.org
Diamond-Blackfan anemia (DBA) is a congenital disorder characterized by the failure of
erythroid progenitor differentiation, severely curtailing red blood cell production. Because …

A BMP-2–triggered in vivo osteo-organoid for cell therapy

K Dai, Q Zhang, S Deng, Y Yu, F Zhu, S Zhang… - Science …, 2023 - science.org
Cell therapies and regenerative medicine interventions require an adequate source of
therapeutic cells. Here, we demonstrate that constructing in vivo osteo-organoids by …

Single-cell analyses and machine learning define hematopoietic progenitor and HSC-like cells derived from human PSCs

A Fidanza, PS Stumpf, P Ramachandran… - Blood, The Journal …, 2020 - ashpublications.org
Hematopoietic stem and progenitor cells (HSPCs) develop in distinct waves at various
anatomical sites during embryonic development. The in vitro differentiation of human …