Translating CRISPR to Practice in the Clinic: Transformative Therapy in Hemoglobinopathies and Emerging Applications in Malignancies.
Hemoglobinopathies provide the first clinically validated delivery model for CRISPR therapeutics, and offers a donor-independent alternative to allogeneic hematopoietic stem-cell transplantation without graft rejection or graft-versus-host disease.
Miza Salim Hammoud, Rabi Hanna
· JCO Oncology Practice · 0 citations