Masoud Nasri
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View article: Flavopiridol restores granulopoiesis in experimental models of severe congenital neutropenia
Flavopiridol restores granulopoiesis in experimental models of severe congenital neutropenia Open
Severe congenital neutropenia (CN) patients require life-long treatment with recombinant human granulocyte colony-stimulating factor (rhG-CSF), but some show no response. We sought to establish a therapy for CN that targets signaling pathw…
View article: Differential transcriptional control of hematopoiesis in congenital and cycling neutropenia patients harboring <i>ELANE</i> mutations
Differential transcriptional control of hematopoiesis in congenital and cycling neutropenia patients harboring <i>ELANE</i> mutations Open
Mutations in the ELANE gene, encoding the neutrophil elastase (NE) protein, are responsible for most CyN cases and approximately 25 % of CN cases. In CN and in CyN, a median of 2.8 % of CD34+ cells were early CD49f+ hematopoietic stem cell…
View article: P1357: MILESTONE (MODIFYING ELANE GOLDBERG–HOGNES BOX TO INHIBIT EXPRESSION), A UNIVERSAL, SAFE AND EFFECTIVE CRISPR/CAS9N-MEDIATED GENOME EDITING STRATEGY FOR ELANE RELATED SEVERE CONGENITAL NEUTROPENIA
P1357: MILESTONE (MODIFYING ELANE GOLDBERG–HOGNES BOX TO INHIBIT EXPRESSION), A UNIVERSAL, SAFE AND EFFECTIVE CRISPR/CAS9N-MEDIATED GENOME EDITING STRATEGY FOR ELANE RELATED SEVERE CONGENITAL NEUTROPENIA Open
Topic: 24. Gene therapy, cellular immunotherapy and vaccination - Biology & Translational Research Background: Severe congenital neutropenia (CN) is an inherited bone marrow failure syndrome. Autosomal-dominant ELANE mutations are the most…
View article: P751: UNRAVELING THE DIFFERENT PATHOMECHANISMS OF CONGENITAL AND CYCLIC NEUTROPENIAS: CYCLIC EXPRESSION OF HEMATOPOIETIC STEM-CELL-SPECIFIC TRANSCRIPTION FACTORS IN CYCLIC NEUTROPENIA
P751: UNRAVELING THE DIFFERENT PATHOMECHANISMS OF CONGENITAL AND CYCLIC NEUTROPENIAS: CYCLIC EXPRESSION OF HEMATOPOIETIC STEM-CELL-SPECIFIC TRANSCRIPTION FACTORS IN CYCLIC NEUTROPENIA Open
Topic: 11. Bone marrow failure syndromes incl. PNH - Biology & Translational Research Background: Severe congenital neutropenia (CN) and cyclic neutropenia (CyN) are disorders of hematopoiesis that differ markedly in disease severity. Muta…
View article: Design of novel granulopoietic proteins by topological rescaffolding
Design of novel granulopoietic proteins by topological rescaffolding Open
Computational protein design is rapidly becoming more powerful, and improving the accuracy of computational methods would greatly streamline protein engineering by eliminating the need for empirical optimization in the laboratory. In this …
View article: NAMPT/SIRT2–mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells
NAMPT/SIRT2–mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells Open
Background: Nicotinamide phosphoribosyltransferase (NAMPT) regulates cellular functions through the protein deacetylation activity of nicotinamide adenine dinucleotide (NAD+)-dependent sirtuins (SIRTs). SIRTs regulate functions of histones…
View article: NAMPT/SIRT2–mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells
NAMPT/SIRT2–mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells Open
Background: Nicotinamide phosphoribosyltransferase (NAMPT) regulates cellular functions through the protein deacetylation activity of nicotinamide adenine dinucleotide (NAD+)-dependent sirtuins (SIRTs). SIRTs regulate functions of histones…
View article: LMO2 activation by deacetylation is indispensable for hematopoiesis and T-ALL leukemogenesis
LMO2 activation by deacetylation is indispensable for hematopoiesis and T-ALL leukemogenesis Open
Key Points LMO2 is deacetylated by the NAMPT/SIRT2 pathway. LMO2 deacetylation is essential for LIM domain binding 1 binding and TAL1 complex activation during hematopoiesis and T-ALL leukemogenesis.
View article: CRISPR/Cas9-mediated <i>ELANE</i> knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patients
CRISPR/Cas9-mediated <i>ELANE</i> knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patients Open
A Autosomal-dominant ELANE mutations are the most common cause of severe congenital neutropenia. Although the majority of congenital neutropenia patients respond to daily granulocyte colony stimulating factor, approximately 15 % do not res…
View article: PF343 CRISPR/CAS9 MEDIATED ELANE KNOCKOUT ENABLES NEUTROPHILIC MATURATION OF HSPCS AND IPSCS OF SEVERE CONGENITAL NEUTROPENIA PATIENTS
PF343 CRISPR/CAS9 MEDIATED ELANE KNOCKOUT ENABLES NEUTROPHILIC MATURATION OF HSPCS AND IPSCS OF SEVERE CONGENITAL NEUTROPENIA PATIENTS Open
Background: Autosomal‐dominant ELANE mutations are the most common cause of severe congenital neutropenia (CN). Although the majority of CN patients respond to daily granulocyte colony stimulating factor (G‐CSF), approximately 15 % do not …
View article: Fluorescent labeling of CRISPR/Cas9 RNP for gene knockout in HSPCs and iPSCs reveals an essential role for GADD45b in stress response
Fluorescent labeling of CRISPR/Cas9 RNP for gene knockout in HSPCs and iPSCs reveals an essential role for GADD45b in stress response Open
CRISPR/Cas9-mediated gene editing of stem cells and primary cell types has several limitations for clinical applications. The direct delivery of ribonucleoprotein (RNP) complexes consisting of Cas9 nuclease and guide RNA (gRNA) has improve…