Bernardo Maria Ester

Bernardo Maria Ester serves as Associate Professor of Pediatrics at Vita-Salute San Raffaele University's School of Medicine, where he holds multiple leadership roles including Member of the Steering Committee for the PhD Program in Molecular Medicine and Deputy Director of its 'Cell and Gene Therapy' curriculum. His clinical work centers at IRCCS San Raffaele Hospital as Head of the Pediatric Immuno-hematology Unit and Coordinator of the Pediatric Allogeneic Transplant Area.

• PhD in Biological Characterization and Clinical Application of Human Mesenchymal Stem Cells (Leiden University, 2010)
• Specialization in Pediatrics (University of Pavia, 2005, cum laude)
• Degree in Medicine and Surgery (University of Pavia, 2000, cum laude)

His research bridges clinical practice and laboratory investigation with focus on gene therapy for metabolic disorders and hematopoietic stem cell transplantation. As a translational physician-scientist, he pioneered the first-in-human lentiviral gene therapy trial for Mucopolysaccharidosis Type I (Hurler variant) serving as Principal Investigator. His work spans stem cell biology, immunomodulation using mesenchymal stromal cells, and therapeutic development for rare pediatric diseases including ADA-SCID and Metachromatic Leukodystrophy.

Analysis of his 15 most recent publications reveals consistent focus on ex-vivo gene therapy (72% of works), with particular emphasis on mucopolysaccharidoses and lysosomal storage disorders. His clinical research examines long-term outcomes, skeletal manifestations, and real-world implementation challenges, while basic science contributions explore transgene optimization and immunomodulatory mechanisms.

• AIRC Investigator Grant (2009) for tumor angiogenesis research
• Ministry of Health Young Researcher Grant (2008)
• Telethon Foundation Core Grant as Principal Investigator (2016-2021)
• European Commission Framework Program participation (Allostem, RETHRIM)

As an educator, he supervises PhD candidates across multiple institutions and mentors 4 pediatric residents annually. His clinical trials portfolio includes national and international studies on gene therapy for ADA-SCID, Wiskott-Aldrich syndrome, and beta-thalassemia. At SR-TIGET (San Raffaele Telethon Institute for Gene Therapy), he leads the Pediatric Clinical Research Unit and serves as Project Leader developing novel cellular therapies.