Advances in stem cell transplantation for Fanconi anemia

While we continue to generate evidence to determine the optimal candidates, timing, and strategies for HCT in FA patients, we must also consider the implications for future treatment modalities like gene therapy/gene editing. The primary objective of all strategies must be to mitigate - or at the very least, not exacerbate - the risk of long-term complications that remain a leading cause of the unacceptably high mortality rate in FA.