A comprehensive review of CRISPR-Cas9 and its evolved variants — including base editors, prime editors, and CRISPR activation/interference systems — highlighting the mechanistic underpinnings of each tool and their expanding utility in disease modelling and therapeutic development. The chapter surveys delivery strategies, off-target effects, and the regulatory landscape governing clinical applications. Recent milestones in the treatment of haemoglobinopathies and other monogenic disorders are discussed in the context of the broader gene editing revolution.

Medical Science
CRISPR-Based Genome Editing: Mechanisms, Innovations, and Therapeutic Frontiers
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