Review
Open access
Jul 2026
Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier
This review examines the central role of conditioning in enabling durable engraftment of gene-modified hematopoietic stem cells, emphasizing the narrow therapeutic window between insufficient niche clearance and excessive toxicity.
Akshay Sharma, S. Corbacioglu
· Blood Advances · 0 citations