She Was Born Without an Immune System. Gene Therapy Saved Her Life.
The New York Times • • health
Key Points:
- Cora Oakley, born with severe combined immunodeficiency (SCID), a rare genetic disorder causing an almost nonexistent immune system, died from a common cold due to her condition.
- SCID, also known as "bubble boy disease," typically requires a bone marrow transplant from a perfectly matched sibling, but Cora had no siblings, making treatment options limited.
- In 2017, Cora received a promising gene therapy trial aimed at fixing her stem cells, specifically targeting ADA-SCID, one of the few SCID types treatable with this approach.
- Before gene therapy, survival for SCID patients depended heavily on early diagnosis and access to bone marrow transplants, which often came with severe side effects.
