Pediatric Immunohematology and Gene Therapy

Gene therapy is the most prominent among advanced therapies, representing a revolutionary approach in modern medicine. At its core lies a visionary idea: addressing the root cause of a disease by providing malfunctioning cells with a functional version of a gene. This innovative strategy aims to treat diseases by acting directly on their genetic basis, introducing a corrected copy of a defective gene or an alternative gene capable of compensating for its dysfunction.

Among the primary tools used to deliver genetic material are viral vectors: viruses stripped of their harmful content and rendered harmless, then repurposed as carriers for therapeutic genetic material. These vectors enable precise delivery of genes into target cells, offering a pathway to address conditions previously deemed incurable. This transformative technique has shown promise for a range of conditions, including inherited disorders such as hemophilia and acquired diseases like certain cancers. By tackling the problem at its genetic root, gene therapy offers hope for patients with diseases that have long eluded effective treatment.

At the forefront of this field, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a global leader. Founded by Fondazione Telethon and Ospedale San Raffaele, SR-Tiget combines clinical expertise with groundbreaking research to advance gene and cell therapies. The institute focuses on translating scientific discoveries from preclinical studies to early-phase clinical trials, developing innovative therapies that are safe and effective.

SR-Tiget has pioneered the use of lentiviral vectors derived from the HIV virus, repurposing these as powerful tools for delivering genetic therapies. Its research priorities include refining hematopoietic stem cell (HSC) gene therapy for rare diseases, advancing gene-editing technologies, and exploring novel strategies to regulate immune responses. These efforts are not only expanding the possibilities of gene therapy but are also shaping the future of personalized medicine.