Malte Ritter
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View article: Ex Vivo Evaluation of Poly(Solketal Acrylate) Nanoparticles for Intravitreal Drug Delivery to the Posterior Eye Segment
Ex Vivo Evaluation of Poly(Solketal Acrylate) Nanoparticles for Intravitreal Drug Delivery to the Posterior Eye Segment Open
Owing the complex anatomy and multiple physiological barriers, delivering drugs to the posterior segment of the eye remains challenging. While intravitreal injection can improve drug delivery, the diffusion of therapeutics through the vitr…
View article: Tuning of G-CSFR signaling by de novo-designed agonists
Tuning of G-CSFR signaling by de novo-designed agonists Open
Enhancing cytokine-based therapies by systematically tuning how an agonist associates its receptor is emerging as a powerful new concept in drug discovery. Here, we report the design and characterization of agonists that tune granulocyte-c…
View article: A new severe congenital neutropenia syndrome associated with autosomal recessive <i>COPZ1</i> mutations
A new severe congenital neutropenia syndrome associated with autosomal recessive <i>COPZ1</i> mutations Open
We have identified a new inherited bone marrow failure syndrome with severe congenital neutropenia (CN) caused by autosomal recessive mutations in the coatomer protein complex I (COPI) subunit zeta 1 (COPZ1) gene. A stop-codon COPZ1 mutati…
View article: Modulating G-CSFR Signaling Using <i>De Novo</i> Designed Cytokines
Modulating G-CSFR Signaling Using <i>De Novo</i> Designed Cytokines Open
Tightly modulating cytokine receptors, which naturally decode complex patterns of downstream signaling, is key to treating a wide range of diseases. De novo protein design is a powerful approach for creating ligands that can modulate the r…
View article: Expanding the genetic landscape of congenital neutropenia: <i>CXCR2</i> mutations in three families revealed through whole exome sequencing
Expanding the genetic landscape of congenital neutropenia: <i>CXCR2</i> mutations in three families revealed through whole exome sequencing Open
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View article: Tuning of granulopoietic signaling by<i>de novo</i>designed agonists
Tuning of granulopoietic signaling by<i>de novo</i>designed agonists Open
Enhancing cytokine-based therapies by systematically tuning how an agonist associates its receptor is emerging as a powerful new concept in drug discovery. Here, we report the design and characterization of agonists that tune the granulocy…
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: 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…