Unlocking the potential: advancements and applications of gene therapy in severe disorders
Introduction Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health.Background Over the past few decades, researchers h...
Saved in:
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Taylor & Francis Group
2025-12-01
|
| Series: | Annals of Medicine |
| Subjects: | |
| Online Access: | https://www.tandfonline.com/doi/10.1080/07853890.2025.2516697 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1850100688445505536 |
|---|---|
| author | Rahul G. Ingle Gehan M. Elossaily Mohd Nazam Ansari Shivani Makhijani |
| author_facet | Rahul G. Ingle Gehan M. Elossaily Mohd Nazam Ansari Shivani Makhijani |
| author_sort | Rahul G. Ingle |
| collection | DOAJ |
| description | Introduction Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health.Background Over the past few decades, researchers have been working on gene therapies, and currently, terrible dreams have come true. To date, the Food and Drug Administration (FDA) has approved multiple gene therapies such as Kynamro for familial hypercholesterolaemia, Exondys51 for duchenne muscular dystrophy, Spinraza for spinal muscular atrophy, etc., rest for cancer, infectious diseases, and rare diseases.Discussion The authors have summarized recent advances in gene therapy, its background, molecular basis (e.g. viral and non-viral vectors), gene-editing techniques (e.g. CRISPR/Cas9, TALEN, ZFN), and its foremost applications in severe disorders, such as cancer, monogenic disorders (e.g. spinal muscular atrophy), polygenic disorders (e.g. autism), neurogenic disorders (e.g. Parkinson disease and Alzheimer’s disease), and infectious diseases (e.g. HIV).Challenges In addition, we explored the major challenges faced by gene therapies during targeted delivery, immunogenicity, efficacy, and safety.Conclusion To date, most of the promising approaches, such as different vectors, target cell populations, and both in vivo and ex vivo have paved the foundation for applications of gene therapies. Additionally, advances in enhancing the immune system that would certainly lower the healthcare costs. This review highlights the translatory potential of gene therapy in revolutionizing the treatment landscape for severe disorders. |
| format | Article |
| id | doaj-art-635ea004914640dfa3c497f2bc662a22 |
| institution | DOAJ |
| issn | 0785-3890 1365-2060 |
| language | English |
| publishDate | 2025-12-01 |
| publisher | Taylor & Francis Group |
| record_format | Article |
| series | Annals of Medicine |
| spelling | doaj-art-635ea004914640dfa3c497f2bc662a222025-08-20T02:40:14ZengTaylor & Francis GroupAnnals of Medicine0785-38901365-20602025-12-0157110.1080/07853890.2025.2516697Unlocking the potential: advancements and applications of gene therapy in severe disordersRahul G. Ingle0Gehan M. Elossaily1Mohd Nazam Ansari2Shivani Makhijani3Datta Meghe College of Pharmacy, Datta Meghe Institute of Higher Education & Research (DMIHER), Deemed to be University, Wardha, IndiaDepartment of Basic Medical Sciences, College of Medicine, AlMaarefa University, Riyadh, Saudi ArabiaDepartment of Pharmacology & Toxicology, College of Pharmacy, Prince Sattam Bin Abdulaziz University, Alkharj, Saudi ArabiaDatta Meghe College of Pharmacy, Datta Meghe Institute of Higher Education & Research (DMIHER), Deemed to be University, Wardha, IndiaIntroduction Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health.Background Over the past few decades, researchers have been working on gene therapies, and currently, terrible dreams have come true. To date, the Food and Drug Administration (FDA) has approved multiple gene therapies such as Kynamro for familial hypercholesterolaemia, Exondys51 for duchenne muscular dystrophy, Spinraza for spinal muscular atrophy, etc., rest for cancer, infectious diseases, and rare diseases.Discussion The authors have summarized recent advances in gene therapy, its background, molecular basis (e.g. viral and non-viral vectors), gene-editing techniques (e.g. CRISPR/Cas9, TALEN, ZFN), and its foremost applications in severe disorders, such as cancer, monogenic disorders (e.g. spinal muscular atrophy), polygenic disorders (e.g. autism), neurogenic disorders (e.g. Parkinson disease and Alzheimer’s disease), and infectious diseases (e.g. HIV).Challenges In addition, we explored the major challenges faced by gene therapies during targeted delivery, immunogenicity, efficacy, and safety.Conclusion To date, most of the promising approaches, such as different vectors, target cell populations, and both in vivo and ex vivo have paved the foundation for applications of gene therapies. Additionally, advances in enhancing the immune system that would certainly lower the healthcare costs. This review highlights the translatory potential of gene therapy in revolutionizing the treatment landscape for severe disorders.https://www.tandfonline.com/doi/10.1080/07853890.2025.2516697Apoptosiscancergenetic disordersgene therapyinfectious diseasesneurodegenerative disorders |
| spellingShingle | Rahul G. Ingle Gehan M. Elossaily Mohd Nazam Ansari Shivani Makhijani Unlocking the potential: advancements and applications of gene therapy in severe disorders Annals of Medicine Apoptosis cancer genetic disorders gene therapy infectious diseases neurodegenerative disorders |
| title | Unlocking the potential: advancements and applications of gene therapy in severe disorders |
| title_full | Unlocking the potential: advancements and applications of gene therapy in severe disorders |
| title_fullStr | Unlocking the potential: advancements and applications of gene therapy in severe disorders |
| title_full_unstemmed | Unlocking the potential: advancements and applications of gene therapy in severe disorders |
| title_short | Unlocking the potential: advancements and applications of gene therapy in severe disorders |
| title_sort | unlocking the potential advancements and applications of gene therapy in severe disorders |
| topic | Apoptosis cancer genetic disorders gene therapy infectious diseases neurodegenerative disorders |
| url | https://www.tandfonline.com/doi/10.1080/07853890.2025.2516697 |
| work_keys_str_mv | AT rahulgingle unlockingthepotentialadvancementsandapplicationsofgenetherapyinseveredisorders AT gehanmelossaily unlockingthepotentialadvancementsandapplicationsofgenetherapyinseveredisorders AT mohdnazamansari unlockingthepotentialadvancementsandapplicationsofgenetherapyinseveredisorders AT shivanimakhijani unlockingthepotentialadvancementsandapplicationsofgenetherapyinseveredisorders |