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 …

Integrating micro and nano technologies for cell engineering and analysis: toward the next generation of cell therapy workflows

P Mukherjee, SH Park, N Pathak, CA Patino, G Bao… - ACS …, 2022 - ACS Publications
The emerging field of cell therapy offers the potential to treat and even cure a diverse array
of diseases for which existing interventions are inadequate. Recent advances in micro and …

Targeting fetal hemoglobin expression to treat β hemoglobinopathies

MH Steinberg - Expert Opinion on Therapeutic Targets, 2022 - Taylor & Francis
Introduction Sickle cell disease and β thalassemia are the principal β hemoglobinopathies.
The complex pathophysiology of sickle cell disease is initiated by sickle hemoglobin …

The dark side of stemness–the role of hematopoietic stem cells in development of blood malignancies

J Filipek-Gorzała, P Kwiecińska, A Szade… - Frontiers in …, 2024 - frontiersin.org
Hematopoietic stem cells (HSCs) produce all blood cells throughout the life of the organism.
However, the high self-renewal and longevity of HSCs predispose them to accumulate …