From haematopoietic stem cells to complex differentiation landscapes

E Laurenti, B Göttgens - Nature, 2018 - nature.com
The development of mature blood cells from haematopoietic stem cells has long served as a
model for stem-cell research, with the haematopoietic differentiation tree being widely used …

[HTML][HTML] Hematopoietic stem cell metabolism during development and aging

A Nakamura-Ishizu, K Ito, T Suda - Developmental cell, 2020 - cell.com
Cellular metabolism in hematopoietic stem cells (HSCs) is an area of intense research
interest, but the metabolic requirements of HSCs and their adaptations to their niches during …

Chemically defined cytokine-free expansion of human haematopoietic stem cells

M Sakurai, K Ishitsuka, R Ito, AC Wilkinson, T Kimura… - Nature, 2023 - nature.com
Haematopoietic stem cells (HSCs) are a rare cell type that reconstitute the entire blood and
immune systems after transplantation and can be used as a curative cell therapy for a variety …

On-chip recapitulation of clinical bone marrow toxicities and patient-specific pathophysiology

DB Chou, V Frismantas, Y Milton, R David… - Nature biomedical …, 2020 - nature.com
The inaccessibility of living bone marrow (BM) hampers the study of its pathophysiology
under myelotoxic stress induced by drugs, radiation or genetic mutations. Here, we show …

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

A Lattanzi, J Camarena, P Lahiri, H Segal… - Science translational …, 2021 - science.org
Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …

Expansion of primitive human hematopoietic stem cells by culture in a zwitterionic hydrogel

T Bai, J Li, A Sinclair, S Imren, F Merriam, F Sun… - Nature medicine, 2019 - nature.com
The ability to expand hematopoietic stem and progenitor cells (HSPCs) ex vivo is critical to
fully realize the potential of HSPC-based therapies. In particular, the application of clinically …

Harnessing matrix stiffness to engineer a bone marrow niche for hematopoietic stem cell rejuvenation

X Zhang, D Cao, L Xu, Y Xu, Z Gao, Y Pan, M Jiang… - Cell stem cell, 2023 - cell.com
Hematopoietic stem cell (HSC) self-renewal and aging are tightly regulated by paracrine
factors from the bone marrow niche. However, whether HSC rejuvenation could be achieved …

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

DP Dever, RO Bak, A Reinisch, J Camarena… - Nature, 2016 - nature.com
The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by
mutations in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene …

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

S Ferrari, A Jacob, S Beretta, G Unali, L Albano… - Nature …, 2020 - nature.com
Targeted gene editing in hematopoietic stem cells (HSCs) is a promising treatment for
several diseases. However, the limited efficiency of homology-directed repair (HDR) in …

Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

S Ferrari, E Valeri, A Conti, S Scala, A Aprile… - Cell Stem Cell, 2023 - cell.com
The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …