Over 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

Bioanalysis

通过对生物样品中的异生物质和生物进行选择性、灵敏和可重复分析获得的可靠数据是每个成功的药物开发计划的基础和关键部分。同样的原则也适用于许多其他研究领域，例如法医学、毒理学和运动兴奋剂检测。生物分析领域结合了复杂的技术，将样品制备和高级分离与 MS 和 NMR 检测系统、自动化和机器人技术相结合。监管机构制定的方法开发和验证标准越来越多地定义了速度和质量之间的界限。生物分析是一门进步的学科，未来拥有许多令人兴奋的机会来进一步减少样品量、分析成本和环境影响，以及提高灵敏度、特异性、准确性、效率、测定通量、数据质量、数据处理和处理。 《生物分析》杂志侧重于用于检测或定量研究人类或动物生物样本中分析物的技术和方法。生物分析鼓励提交描述前瞻性应用的文章，包括生物传感器、微流体、小型化分析设备以及新的连字符和多维技术。生物分析以简洁、一目了然的文章格式提供基本信息。该领域的主要进展由国际专家报告和分析，为现代生物分析家提供了一个权威但易于访问的论坛。

Reliable data obtained through selective, sensitive, and reproducible analysis of xenobiotics and biomolecules in biological samples is fundamental to any successful drug development program. This principle also applies to many other research fields, such as forensic science, toxicology, and doping detection. The field of bioanalysis integrates complex technologies, combining sample preparation and advanced separation with mass spectrometry (MS) and nuclear magnetic resonance (NMR) detection systems, automation, and robotics. Regulatory agencies are increasingly defining the boundaries between speed and quality through method development and validation standards. Bioanalysis is an evolving discipline with many exciting opportunities in the future to further reduce sample volume, analysis costs, and environmental impact, while enhancing sensitivity, specificity, accuracy, efficiency, throughput, data quality, data processing, and handling capabilities. The journal "Bioanalysis" focuses on technologies and methods used for the detection or quantification of analytes in human or animal biological samples. Bioanalysis encourages the submission of articles describing forward-looking applications, including biosensors, microfluidics, miniaturized analytical devices, and novel hyphenated and multidimensional techniques. The journal presents essential information in a concise and clear article format, with major advancements in the field reported and analyzed by international experts, providing a credible yet accessible forum for modern bioanalysts.

生物样品中异生物质和生物的选择性、灵敏和可重复分析所获得的可靠数据，是成功药物开发计划的基础和关键部分。这一原则同样适用于法医学、毒理学和运动兴奋剂检测等多个研究领域。生物分析领域结合了复杂的技术，将样品制备、高级分离与质谱（MS）和核磁共振（NMR）检测系统、自动化和机器人技术相结合。监管机构制定的方法开发和验证标准，越来越多地定义了速度与质量之间的界限。生物分析是一门不断进步的学科，未来有许多令人兴奋的机会，可以进一步减少样品量、分析成本和环境影响，同时提高灵敏度、特异性、准确性、效率、测定通量、数据质量、数据处理和处理能力。《Bioanalysis》杂志专注于用于检测或定量研究人类或动物生物样本中分析物的技术和方法。生物分析鼓励提交描述前瞻性应用的文章，包括生物传感器、微流体、小型化分析设备以及新的连锁和多维技术。该杂志以简洁、一目了然的文章格式提供基本信息，领域内的主要进展由国际专家报告和分析，为现代生物分析家提供了一个权威但易于访问的论坛。

Category	Quartile
医学	4
医学, 生化研究方法	4
医学, 分析化学	4

Review on the bioanalysis of non-virus-based gene therapeutics

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Journal Info

Category	Quartile
CHEMISTRY, ANALYTICAL	3