OMER ATTYA

My research focuses on hematopoietic stem cell (HSC) biology, genome engineering, and the development of innovative strategies to improve the safety and efficacy of HSC-based gene therapies. I am currently a Project Leader at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, in the laboratory of Prof. Luigi Naldini. My research program primarily aims to develop non-genotoxic conditioning strategies that could replace or reduce the chemotherapy and radiotherapy currently required to enable the engraftment of genetically modified cells. These treatments are associated with significant toxicity and represent a major obstacle to extending gene therapy to a broader range of patients and diseases. In particular, I investigate the molecular mechanisms governing HSC mobilization, retention, trafficking, and homing. Through genome-wide screening, CRISPR technologies, and transient mRNA-based engineering, my group identifies and modulates factors that promote the release of HSCs from the bone marrow and improve the engraftment of transplanted cells. A central component of this work is the development of mobilization-based transplantation approaches designed to create temporary space within the bone marrow without requiring myeloablative conditioning. My research also includes strategies to selectively protect transplanted HSCs from antibodies or other agents targeting specific cell-surface receptors, as well as the use of lipid nanoparticles and RNA-based platforms to transiently modify hematopoietic cells or the bone marrow microenvironment. These approaches are evaluated in preclinical models of inherited immunodeficiencies and other hematological disorders, with particular emphasis on safety, clinical translatability, and their potential application in gene therapy. My scientific background also includes research in pluripotent stem cells, genome engineering, and cerebral organoids, conducted during my doctoral training at the Whitehead Institute for Biomedical Research and the Massachusetts Institute of Technology. During this period, I used human cellular models to investigate brain development and the mechanisms underlying neurological disorders. The overall objective of my research is to translate fundamental discoveries in stem cell biology into innovative, less toxic, and more accessible therapeutic strategies, thereby contributing to the clinical development and broader implementation of gene therapies for genetic and hematological diseases.