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Review
. 2025 Jul;144(7):695-713.
doi: 10.1007/s00439-025-02757-7. Epub 2025 Jun 18.

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

Shireen Nishad  1 , Dipali Dongare  1 , Sayani Saha  1 , Raskar Dhanashri Anil  1 , Nidhi Srivastava  1 , Abhishek Dey  2
Affiliations
Review

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

Shireen Nishad et al. Hum Genet. 2025 Jul.

Abstract

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.

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Conflict of interest statement

Declarations. Conflict of interest: The authors declare no competing interests. Ethics approval and consent: No human or animal experiments were performed by the authors for this review article. Consent for publication: All the authors who are part of this manuscript have given their approval to the final version of this manuscript for publication.

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