CRISPR-Cas9 Gene Therapy in Cancer Immunotherapy and Inherited Retinal Disease: A Systematic Review

This review directly addresses the scope of Genome Editing in Cancer and Immunology by synthesizing clinical and translational evidence on CRISPR-Cas9 gene therapy across two of its most advanced therapeutic domains: cancer immunotherapy and inherited retinal disease. In the oncology context, we review first-in-human trials of CRISPR-edited autologous and allogeneic CAR-T cells, tumor-infiltrating lymphocyte engineering, and checkpoint gene disruption strategies, including the landmark Stadtmauer et al. (2020) multiplex editing study and the CISH-knockout TIL trial (Lou et al., 2025). We further contextualize these findings within the broader landscape of delivery platforms, off-target safety, immune responses to Cas9, and regulatory milestones including the FDA approval of Casgevy. This review provides a rigorous, PRISMA-compliant synthesis of the current evidence base, serving as a foundational reference for researchers developing next-generation genome editing strategies for cancer immunotherapy.