Schneider Children Hospital
Pediatric-hematology and hemato-oncology research labs are part of Rina Zaizov Division in Schneider Children’s Medical Center, Israel. Both clinical services and labs are headed by Prof Shai Izraeli, a proximity that allows unique interaction for translational research from bed to bench and back to the clinic. The research laboratories focus on inherited disorders of blood production (“bone marrow failure syndromes”) and on pediatric cancer, especially blood cancers.
Our vision is to use gene editing and therapies towards cure of blood disorders. We study induced pluripotent stem-cells (iPSCs) generated from congenital neutropenia patients that recapitulate the myeloid differentiation arrest found in neutropenia. We aim to identify phenotypic differences between severe congenital or cyclic neutropenia and how these disorders undergo malignant transformation, in order to determine the correct timing for intervention by bone marrow transplantation or by gene therapy through advanced gene editing.
Partner's Genome Editing vision
Using CRISPR/Cas9 gene editing system on iPSCs for the introduction of somatic mutations found in neutropenia patients. Gene editing is used to make a hematological oncology models in cell lines to understand disease mechanism and optional repair.
Partner's activity within the consortium
Schneider group is contributing by CRISPR gene-editing performing in our cellular models and sequencing of these editing events. Our results are used for machine learning to improve the targeting efficiency and to eliminate off-target effects.