Gene therapy using haematopoietic stem and progenitor cells

G Ferrari, AJ Thrasher, A Aiuti - Nature Reviews Genetics, 2021 - nature.com
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

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 …

[HTML][HTML] The stem cell niches in bone

T Yin, L Li - The Journal of clinical investigation, 2006 - Am Soc Clin Investig
The stem cell niche is composed of a specialized population of cells that plays an essential
role in regulating adult stem cell self-renewal and differentiation. In adults, osteoblasts …

[HTML][HTML] Current understanding of stem cell mobilization: the roles of chemokines, proteolytic enzymes, adhesion molecules, cytokines, and stromal cells

T Lapidot, I Petit - Experimental hematology, 2002 - Elsevier
Mobilization of hematopoietic stem and progenitor cells from the bone marrow into the
circulation by repetitive, daily stimulations with G-CSF alone, or in combination with …

The prognostic and functional role of microRNAs in acute myeloid leukemia

G Marcucci, K Mrózek, MD Radmacher… - Blood, The Journal …, 2011 - ashpublications.org
Expression of microRNAs, a new class of noncoding RNAs that hybridize to target
messenger RNA and regulate their translation into proteins, has been recently demonstrated …

HMG-CoA reductase inhibitors reduce senescence and increase proliferation of endothelial progenitor cells via regulation of cell cycle regulatory genes

B Assmus, C Urbich, A Aicher, WK Hofmann… - Circulation …, 2003 - Am Heart Assoc
Endothelial progenitor cells (EPCs) play an important role in postnatal neovascularization of
ischemic tissue. Ex vivo expansion of EPCs might be useful for potential clinical cell therapy …

[HTML][HTML] Hematopoietic reconstitution dynamics of mobilized-and bone marrow-derived human hematopoietic stem cells after gene therapy

S Scala, F Ferrua, L Basso-Ricci, F Dionisio… - Nature …, 2023 - nature.com
Mobilized peripheral blood is increasingly used instead of bone marrow as a source of
autologous hematopoietic stem/progenitor cells for ex vivo gene therapy. Here, we present …

Stem cell mobilization

MH Cottler-Fox, T Lapidot, I Petit… - ASH Education …, 2003 - ashpublications.org
Successful blood and marrow transplant (BMT), both autologous and allogeneic, requires
the infusion of a sufficient number of hematopoietic progenitor/stem cells (HPCs) capable of …

PAR1 signaling regulates the retention and recruitment of EPCR-expressing bone marrow hematopoietic stem cells

S Gur-Cohen, T Itkin, S Chakrabarty, C Graf, O Kollet… - Nature medicine, 2015 - nature.com
Retention of long-term repopulating hematopoietic stem cells (LT-HSCs) in the bone marrow
is essential for hematopoiesis and for protection from myelotoxic injury. We report that …

Stem cells for the treatment of spinal cord injury

M Coutts, HS Keirstead - Experimental neurology, 2008 - Elsevier
This article reviews stem cell-based strategies for spinal cord injury repair, and practical
issues concerning their translation to the clinic. Recent progress in the stem cell field …