Skip to search formSkip to main contentSkip to account menu

CRISPR/Cas9-mediated ELANE knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patients

Observations suggest that ex vivo CRISPR/Cas9 RNP based ELANE knockout of patients’ primary hematopoietic stem and progenitor cells followed by autologous transplantation may be an alternative therapy for congenital neutropenia.
FSF (opens in a new tab)

LMO2 activation by deacetylation is indispensable for hematopoiesis and T-ALL leukemogenesis.

It is demonstrated that LMO2 is activated by deacetylation on lysine 74 and 78 via the nicotinamide phosphoribosyltransferase (NAMPT)/sirtuin 2 (SIRT2) pathway, which may provide new therapeutic possibilities in T-ALL and may contribute to the development of new methods for in vitro generation of blood cells.
PubMed (opens in a new tab)

Production, purification and titration of a lentivirus-based vector for gene delivery purposes

The applicability of this lentivirus-based vector for the efficient transduction of dividing and non-dividing cells, including HEK-293T, CHO, HepG2, MCF-7, MEFs and Jurkat cell lines is demonstrated.
Springer Nature (opens in a new tab)

Fluorescent labeling of CRISPR/Cas9 RNP for gene knockout in HSPCs and iPSCs reveals an essential role for GADD45b in stress response.

It is found that GADD45B is indispensable for DNA damage protection and survival in stem cells, and an easy and efficient protocol of DNA-free gene editing of hard-to-target transcripts and enrichment of gene-modified cells that are generally difficult to transfect is described.
PubMed (opens in a new tab)

The chemokine CXCL14 mediates platelet function and migration via direct interaction with CXCR4.

The results reveal CXCL14 as a novel platelet-derived chemokine that is involved in thrombus formation and platelet migration and the CXCR4 interaction could offer novel therapeutic strategies to control thrombosis and inflammation in inflammatory diseases such as atherosclerosis or organ ischemia.
PubMed (opens in a new tab)

CRISPR/Cas9 Genome Editing of Human-Induced Pluripotent Stem Cells Followed by Granulocytic Differentiation.

A stepwise protocol of safe, efficient, and selection-free CRISPR/Cas9-mediated gene correction or knockout in human iPSCs followed by 3D spin-embryoid body (EB)-based hematopoietic/neutrophilic iPSC-differentiation is described.
PubMed (opens in a new tab)

NAMPT/SIRT2–mediated activation of LMO2 by deacetylation is indispensable for hematopoiesis

Publisher (opens in a new tab)

NAMPT-mediated LMO2 deacetylation is indispensable for hematopoiesis and T-ALL leukemogenesis

Thieme (opens in a new tab)

Differential transcriptional control of hematopoiesis in congenital and cycling neutropenia patients harboring ELANE mutations.

In CyN and in CN patients, CD49f+ cells failed to express mRNA levels of HSC-specific transcription factors mentioned above, and rescue of C/EBP//expression in CN restored granulopoiesis.
PDF (opens in a new tab)

CRISPR/Cas9 Knock-in HL60 Cells Closely Simulate Cellular and Functional Abnormalities of ELANE associated Neutropenia; Phenotype Rescue with MK-0339 Neutrophil Elastase Inhibitor

It is reported that MK-0339 increases cell survival and myeloid differentiation in cellular models of ELANE associated neutropenia and this work believes CRISPR/Cas9 engineered HL60 cell lines expressing mutant NE are a highly reproducible and reliable cellular model for investigating genetic neutropanias.
Publisher (opens in a new tab)
By clicking accept or continuing to use the site, you agree to the terms outlined in our Privacy Policy (opens in a new tab), Terms of Service (opens in a new tab), and Dataset License (opens in a new tab)