“They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection.”
– Evangelina Vaccaro’s story
Source: UCLA News
Infants able to live normal life after undergoing groundbreaking gene therapy
UCLA researchers have developed a stem cell gene therapy cure for babies born with by a rare and life-threatening immunodeficiency disorder known as ADA-SCID or “bubble baby” disease. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could be deadly.
Now thanks to a Stage 2 clinical trial at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, 30 out of 30 babies have been cured by the trial. The treatment method uses the child’s own cells to create a stem cell gene therapy that safely restores the immune systems in babies that have the immunodeficiency disorder.