SR-TIGET researchers found a cure for bubble boys, like Sebastian
SR-TIGET (San Raffaele Telethon Institute for Gene Therapy) is world leader in gene therapy, helping children and families from all over the world. Many of the children benefitting from SR-TIGET therapies and treatments are American,
such as Sebastian from California diagnosed with ADA-SCID.
ADA-SCID, once known as “bubble boys disease”, is an inherited condition that affects the immune system.
People with ADA-SCID have a reduced or absent immune response which leaves them vulnerable to frequent infections. Sebastian was born in San Francisco in 2002. Before contacting SR-TIGET, his family went through years of battling infections and frequent hospitalization. He was successfully treated on the ADA-SCID gene therapy trial when he was 6, and is now a healthy fifteen year old boy.
The corrective gene therapy to treat patients with ADA-SCID was regulatory approved in 2016 and it’s now available to patients from all over the world.
"It’s hard to imagine life without Sebastian, and because of his gene therapy treatment, I don’t have to. It’s been nine years since he had gene therapy treatment and his life today is more normal than I could have ever dreamed.” said Sebastian’s mom, Lynette. “This did not happen on its own. This is thanks to the hard work and research of Prof. Aiuti and his colleagues at SR-TIGET. We could not be more grateful."