The Evolution of CRISPR-Cas9 Applications in Gene Therapy for Rare Monogenic Diseases: Current Progress and Future Directions
DOI:
https://doi.org/10.70088/xvebp625Keywords:
CRISPR-Cas9, Gene Therapy, Monogenic Diseases, Genome Editing, Therapeutic ApplicationsAbstract
CRISPR-Cas9 has emerged as a transformative tool in gene therapy, specially for monogenic diseases. This brushup examines the diachronic development of CRISPR-Cas9 applications, explores its core mechanisms and advancements, and assess its role in addressing inherited disorderliness. Key challenges such as off-target effects, delivery systems, hence and honorable retainer are discussed, alongside succeeding directions including precision editing, novel delivery vectors. And curative scalability. By synthesizing current advancement and figure possibleness; this paper course aims to furnish a understanding of CRISPR-Cas9's voltage in overturn gene therapy for disease.References
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Copyright (c) 2025 Chloe Dubois (Author)
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