Gene Therapy for Cancer
Gene Therapy for Leukemia
Leukemia is where gene therapy became real clinical medicine. Tisagenlecleucel (Kymriah) is FDA-approved for relapsed/refractory B-cell ALL with response rates exceeding 80%. Multiple approved CAR-T products target blood cancers. CRISPR-modified next-generation programs are in active trials. Cytokine release syndrome is the primary managed toxicity โ requires treatment at certified centers.
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