Advances in viral vector-based delivery systems for gene therapy: a comprehensive review

被引：1

作者：

Singh, Kuldeep ^{[1
]}

Jain, Divya ^{[2
]}

Sethi, Pranshul ^{[3
,4
]}

Gupta, Jeetendra Kumar ^{[1
]}

Dubey, Anubhav ^{[5
]}

Noman, Abdullah Al ^{[6
]}

Lal, Shipra ^{[7
]}

Sharma, Pranab Dev ^{[8
]}

Abdallah, Emad M. ^{[9
]}

机构：

[1] GLA Univ, Inst Pharmaceut Res, Dept Pharmacol, Mathura, Uttar Pradesh, India

[2] Uttaranchal Univ, Sch Appl & Life Sci, Dept Microbiol, Dehra Dun, Uttaranchal, India

[3] Shri Venkateshwara Univ, Coll Pharm, Dept Pharmacol, Gajraula 244236, Uttar Pradesh, India

[4] Chitkara Univ, Chitkara Coll Pharm, Rajpura 140401, Punjab, India

[5] Maharana Pratap Coll Pharm, Dept Pharmacol, Kanpur, Uttar Pradesh, India

[6] BRAC Univ, Sch Pharm, Dhaka, Bangladesh

[7] IES Univ, IES Inst Pharm, Bhopal 462044, Madhya Pradesh, India

[8] BRAC Univ, Dept Math & Nat Sci, Biotechnol Program, Dhaka, Bangladesh

[9] Qassim Univ, Coll Sci, Dept Biol, Buraydah 51452, Qassim, Saudi Arabia

来源：

3 BIOTECH | 2025年 / 15卷 / 07期

关键词：

Gene therapy; Viral vectors; Drug development; Health; Cancer drug therapy; ADENOASSOCIATED VIRUS AAV; FACTOR-IX GENE; HUMORAL IMMUNE-RESPONSES; HERPES-SIMPLEX-VIRUS; T-CELL RESPONSES; OPEN-LABEL; ADENOVIRAL VECTORS; INSERTIONAL MUTAGENESIS; NEUTRALIZING ANTIBODIES; TRANSGENE DELIVERY;

D O I：

10.1007/s13205-025-04366-7

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

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.

引用

页数：22

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