CRISPR as a Precision Therapeutic Tool: From HIV and Cancer to One-Time Blood Cures and In Vivo Genome Editing
Year: 2026
Type: article
Abstract: CRISPR-Cas genome editing has transitioned from a bacterial adaptive immune mechanism to a clinically validated therapeutic platform. Its programmable precision enables targeted disruption, correction, or regulation of disease-associated genes. This review critically examines CRISPR application across four major therapeutic domains: HIV eradication strategies, cancer genome engineering and immunotherapy, one-time curative approaches for inherited blood disorders, and in vivo gene editing for direct tissue correction. We discuss molecular mechanisms, delivery systems, clinical translation milestones, safety challenges, and regulatory considerations. While the first CRISPR-based therapies for hematologic diseases have now received regulatory approval, broader in vivo applications remain constrained by delivery efficiency, immunogenicity, and long-term genomic safety. CRISPR's future as a precision "targeted assassin" in medicine will depend on advances in specificity engineering, scalable manufacturing, and equitable global implementation.
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W7225660082
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