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DOI 10.1007/s00439-025-02757-7

Медицинская библиотека · Human genetics · 2025

Current perspectives on gene therapy and its involvement in curing genetic disorders.

Nishad Shireen, Dongare Dipali, Saha Sayani, Anil Raskar Dhanashri, Srivastava Nidhi, Dey Abhishek

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Genomics is revolutionizing medical science, offering transformative potential for the future of medicine. Advances in whole-genome sequencing have deepened our understanding of genome structure and function, paving the way for genomic medicine. The Human Genome Project has been instrumental in identifying genetic variations linked to increased disease risks, such as cancer, enabling genome-based diagnostics and personalized therapeutic strategies. Human genomics research focuses on developing precise therapies to enhance public health and address rare genetic disorders, including Spinal muscular atrophy, Duchenne muscular dystrophy, Parkinson's disease, and Huntington's disease. Cutting-edge gene-editing tools like CRISPR allow precise and targeted modifications with minimal side effects, improving treatment efficacy. By examining the interplay of genetic factors in health and disease, genomics lays the foundation for personalized medicine. This review highlights the impact of genomics on public health and its potential to reshape healthcare through innovative treatment strategies.

Для цитирования: Nishad Shireen, Dongare Dipali, Saha Sayani, Anil Raskar Dhanashri, Srivastava Nidhi, Dey Abhishek. Current perspectives on gene therapy and its involvement in curing genetic disorders.. Human genetics. 2025. DOI: 10.1007/s00439-025-02757-7.

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