Volume-12 Number-3, 2025 / Review Article

CRISPR–Cas9 and In Vivo Genome Editing: Transforming the Future of Therapeutics

Author:
Shifa
Department of Biotechnology, Deen Dayal Upadhyaya Gorakhpur University, Gorakhpur, UP, India
Aqsa Bakhtawar Khan
Department of Biotechnology, Deen Dayal Upadhyaya Gorakhpur University, Gorakhpur, UP, India
Ekta Tiwari
Department of Biotechnology, Deen Dayal Upadhyaya Gorakhpur University, Gorakhpur, UP, India
Nupur Sharma
Department of Biotechnology, Deen Dayal Upadhyaya Gorakhpur University, Gorakhpur, UP, India
Jaya Pandey
Department of Biotechnology, Deen Dayal Upadhyaya Gorakhpur University, Gorakhpur, UP, India
Pawan Kumar Doharey
Department of Biotechnology, Deen Dayal Upadhyaya Gorakhpur University, Gorakhpur, UP, India
Abstract:
One of the finest ways to change genomes is via CRISPR-Cas9. This approach lets us modify DNA exactly and without making any mistakes. We want to emphasize how crucial CRISPR-Cas9 is to human health in this review, especially when it comes to applying it in vivo for diseases like cancer and degenerative ones. Some recent developments include cancer genome editing using patient-derived xenografts (PDXs), treating atherosclerosis with adeno-associated virus (AAV) CRISPR, and repairing photoreceptor degeneration with Cas9-RecA. The study discusses base editing, prime editing, and CRISPR-integrated synthetic biology as potential future directions, along with other Cas systems, the regulatory and ethical landscape, and more subjects. We have added tables and figures to help you compare gene-editing methods with the drug development processes that are already in use. CRISPR is set to alter current medicine by making medicines that weren't possible before. But they are still working out how to deal with off-target consequences, delivery modalities, and germline ethics.
Keywords:
CRISPR, Cas9, Diagnosis, Genome editing, Gene therapy, Strain engineering

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Copyright (c) 2026 Era's Journal of Medical Research

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Download PDF View in DOI 10.24041/ejmr.2025.42