Advances in viral vector-based delivery systems for gene therapy: a comprehensive review
Kuldeep Singha b✉, Divya Jainc d✉, Pranshul Sethie f g h✉, J. K. Guptaa b✉, Anubhav Dubeyi j✉, A. Al Nomank✉, Shipra lall m✉, P. Sharman✉, E. Abdallaho p✉
- aDepartment of Pharmacology, Institute of Pharmaceutical Research, GLA University, Mathura, Uttar Pradesh India
- bDepartment of Pharmacology, Institute of Pharmaceutical Research, GLA University, Mathura, Uttar Pradesh, India
- cDepartment of Microbiology, School of Applied and Life Sciences, Uttaranchal University, Dehradun, Uttarakhand India
- dDepartment of Microbiology, School of Applied and Life Sciences, Uttaranchal University, Dehradun, Uttarakhand, India
- eChitkara College of Pharmacy, Chitkara University, Rajpura, Punjab 140401 India
- fDepartment of Pharmacology, College of Pharmacy, Shri Venkateshwara University, Gajraula, UP 244236 India
- gChitkara College of Pharmacy, Chitkara University, Rajpura, Punjab, 140401, India
- hDepartment of Pharmacology, College of Pharmacy, Shri Venkateshwara University, Gajraula, UP, 244236, India
- iDepartment of Pharmacology, Maharana Pratap College of Pharmacy, Kanpur, Uttar Pradesh India
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Abstract
Gene therapy offers a revolutionary approach for treating genetic and acquired disorders by delivering therapeutic genes to target cells. Viral vectors, engineered for efficient gene delivery, have become indispensable due to their high transduction efficiency, enhanced targeting specificity, and minimized immunogenicity. This review explores recent advancements in viral vector platforms, focusing on intracellular trafficking, transgene expression, and strategies to optimize vector design. Key challenges, including immune responses, insertional mutagenesis, and manufacturing scalability, are critically examined alongside innovative solutions such as hybrid delivery systems and AI-driven capsid design. The impact of these innovations is underscored by the 28 ongoing clinical trials and 16 FDA/EMA-approved gene therapies. In addition, integrating artificial intelligence for vector optimization and CRISPR-based gene correction holds great potential for precision medicine. Future directions include refining non-viral alternatives and improving biomanufacturing for enhanced accessibility. These advancements collectively drive the field toward safer and more effective gene therapy solutions.
TL;DRRecent advancements in viral vector platforms are explored, focusing on intracellular trafficking, transgene expression, and strategies to optimize vector design, to drive the field toward safer and more effective gene therapy solutions. (Semantic Scholar)