Eligibility depends on medical criteria such as diagnosis, transfusion burden, genotype, organ function, and other factors assessed by qualified physicians.

No. Long-term follow-up and periodic monitoring are typically recommended after gene therapy.

The main risks are associated with myeloablative conditioning (a chemotherapy process) and include decreased white blood cells, platelets, and neutrophils, along with nausea, vomiting, and mouth sores. Most reactions are manageable, with Grade 1-2 toxicities being the most common.