Maotian Zhou, Xue Zhang, Huan Yan, Lili Xing, Yi Tao, Liang Shen
Bioanalysis 2024 Dec-DecOver the past years, gene therapeutics have held great promise for treating many inherited and acquired diseases. The increasing number of approved gene therapeutics and developing clinical pipelines demonstrate the potential to treat diseases by modifying their genetic blueprints in vivo. Compared with conventional treatments targeting proteins rather than underlying causes, gene therapeutics can achieve enduring or curative effects via gene activation, inhibition, and editing. However, the delivery of DNA/RNA to the target cell to alter the gene expression is a complex process that involves, crossing numerous barriers in both the extracellular and intracellular environment. Generally, the delivery strategies can be divided into viral-based and non-viral-based vectors. This review summarizes various bioanalysis strategies that support the non-virus-based gene therapeutics research, including pharmacokinetics (PK)/toxicokinetics (TK), biodistribution, immunogenicity evaluations for the gene cargo, vector, and possible expressed protein, and highlights the challenges and future perspectives of bioanalysis strategies in non-virus-based gene therapeutics. This review may provide new insights and directions for the development of emerging bioanalytical methods, offering technical support and a research foundation for innovative gene therapy treatments.
Maotian Zhou, Xue Zhang, Huan Yan, Lili Xing, Yi Tao, Liang Shen. Review on the bioanalysis of non-virus-based gene therapeutics. Bioanalysis. 2024 Dec-Dec;16(23-24):1279-1294
PMID: 39673530
View Full Text