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 …

Chemical reprogramming for cell fate manipulation: methods, applications, and perspectives

J Wang, S Sun, H Deng - Cell Stem Cell, 2023 - cell.com
Chemical reprogramming offers an unprecedented opportunity to control somatic cell fate
and generate desired cell types including pluripotent stem cells for applications in …

Controlling genetic heterogeneity in gene-edited hematopoietic stem cells by single-cell expansion

HJ Becker, R Ishida, AC Wilkinson, T Kimura, MSJ Lee… - Cell stem cell, 2023 - cell.com
Gene editing using engineered nucleases frequently produces unintended genetic lesions
in hematopoietic stem cells (HSCs). Gene-edited HSC cultures thus contain heterogeneous …

Stem cell mutations, associated cancer risk, and consequences for regenerative medicine

LLM Derks, R van Boxtel - Cell Stem Cell, 2023 - cell.com
Mutation accumulation in stem cells has been associated with cancer risk. However, the
presence of numerous mutant clones in healthy tissues has raised the question of what …

VEGF signaling governs the initiation of biliary-mediated liver regeneration through the PI3K-mTORC1 axis

P Cai, R Ni, M Lv, H Liu, J Zhao, J He, L Luo - Cell Reports, 2023 - cell.com
Biliary epithelial cells (BECs) are a potential source to repair the damaged liver when
hepatocyte proliferation is compromised. Promotion of BEC-to-hepatocyte …

Mobilization-based engraftment of haematopoietic stem cells: a new perspective for chemotherapy-free gene therapy and transplantation

D Canarutto, A Omer Javed, G Pedrazzani… - British Medical …, 2023 - academic.oup.com
Introduction In haematopoietic stem cell transplantation (HSCT), haematopoietic stem cells
(HSCs) from a healthy donor replace the patient's ones. Ex vivo HSC gene therapy (HSC …

Generating human bone marrow organoids for disease modeling and drug discovery

AA Olijnik, A Rodriguez-Romera, ZC Wong, Y Shen… - Nature …, 2024 - nature.com
The bone marrow supports and regulates hematopoiesis, responding to physiological
requirements for blood cell production over ontogeny and during pathological challenges …

The genesis of human hematopoietic stem cells

V Calvanese, HKA Mikkola - Blood, 2023 - ashpublications.org
Developmental hematopoiesis consists of multiple, partially overlapping hematopoietic
waves that generate the differentiated blood cells required for embryonic development while …

In vitro human haematopoietic stem cell expansion and differentiation

YK Bozhilov, I Hsu, EJ Brown, AC Wilkinson - Cells, 2023 - mdpi.com
The haematopoietic system plays an essential role in our health and survival. It is comprised
of a range of mature blood and immune cell types, including oxygen-carrying erythrocytes …

Umbilical cord blood and cord tissue banking as somatic stem cell resources to support medical cell modalities

T Nagamura-Inoue, F Nagamura - Inflammation and Regeneration, 2023 - Springer
Human umbilical cord blood (CB) and umbilical cord tissue (UC) are attractive sources of
somatic stem cells for gene and cell therapies. CB and UC can be obtained noninvasively …