Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy

Figures

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  Figure 1. Milestones in the history of CRISPR–Cas technology from its discovery in 1987 until the first FDA-approved gene therapy using CRISPR–Cas9 for genome modification: Ishino [21], Jansen [22], Bolotin [23], Mojica [24], Pourcel [25], Barranougou [26], Deltcheva [27], Jinek [1], Cong [28], Shmakov [29], Cyranoski [30].

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  Figure 2. CTX001 mechanism of action. (A) BCL11A promotes βs-globin expression while suppressing γ-globin expression, resulting in HbS production and the formation of crescent-shaped cells. (B) Knockdown of BCL11A, achieved by introducing a cut at the binding region of transcription factors GATA1/TAL1, enables γ-globin gene expression, leading to HbF production and the development of healthy erythrocytes.