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Examples of Gene-Editing Strategies in Current Clinical Trials
(A) In cancer, gene-editing targets PD-1 or CD7 in T cells to enhance immune responses.
(B) In patients with HIV, CCR5 is targeted in HSPCs to prevent HIV entry.
(C) In β-thalassemia and sickle cell disease, induction of fetal hemoglobin or correction of adult hemoglobin in HSPCs is used.
(D) AAV-mediated gene editing in the liver provides circulating enzymes in lysosomal storage diseases.
