Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy

Affiliations

• ¹ San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) IRCCS San Raffaele Scientific Institute, Milan, Italy. Electronic address: [email protected].
• ² San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita - Salute San Raffaele University Medical School, Milan, Italy.
• ³ UCL Great Ormond Street Institute of Child Health, London, UK; Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Trust, London, UK.
• ⁴ Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA.
• ⁵ San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita - Salute San Raffaele University Medical School, Milan, Italy; Pediatric Immunohematology and BMT, San Raffaele Hospital, Milan, Italy.