合成基因/遗传电路的治疗应用:专利审查。
Therapeutic applications of synthetic gene/genetic circuits: a patent review.
发表日期:2024
作者:
Diego C Carneiro, Vinícius P C Rocha, Patrícia K F Damasceno, Josiane D V Barbosa, Milena B P Soares
来源:
BIOSENSORS & BIOELECTRONICS
摘要:
目前许多基因工程治疗方法的一个显着局限性是它们对其治疗效果的强度、时间或细胞环境的控制有限。合成基因/遗传电路是合成生物学方法,可以控制特定 DNA、RNA 或蛋白质的生成、转化或消耗,并提供对基因表达和细胞行为的精确控制。它们可以被设计为通过仔细选择启动子、阻遏子和其他遗传成分来执行逻辑操作。在 Espacenet 中进行专利检索,选出 38 项专利,其中包含 15 个最常见的国际分类。专利实施例分为治疗分子递送、传染病治疗、癌症治疗、出血治疗和代谢紊乱治疗的应用。描述了选定遗传电路的逻辑门,以全面展示其治疗应用。可以定制合成基因电路,以精确控制治疗干预,从而实现针对个体患者需求的个性化治疗,提高治疗效果并最大限度地减少副作用。它们可以是高度灵敏的生物传感器,通过准确监测各种生物标志物或病原体并适当合成治疗分子来提供实时治疗。合成基因电路还可能促进先进再生疗法的发展,以及产生按需生物活性分子的可植入生物设备。然而,这项技术面临着商业盈利能力的挑战。遗传电路设计需要针对特定应用进行调整,并且可能存在多种缺点,例如来自多个调节器的毒性、同源重组、上下文依赖性、资源过度使用和环境变异性。版权所有 © 2024 Carneiro、Rocha、Damasceno、Barbosa 和 Soares。
A significant limitation of numerous current genetic engineering therapy approaches is their limited control over the strength, timing, or cellular context of their therapeutic effect. Synthetic gene/genetic circuits are synthetic biology approaches that can control the generation, transformation, or depletion of a specific DNA, RNA, or protein and provide precise control over gene expression and cellular behavior. They can be designed to perform logical operations by carefully selecting promoters, repressors, and other genetic components. Patent search was performed in Espacenet, resulting in 38 selected patents with 15 most frequent international classifications. Patent embodiments were categorized into applications for the delivery of therapeutic molecules, treatment of infectious diseases, treatment of cancer, treatment of bleeding, and treatment of metabolic disorders. The logic gates of selected genetic circuits are described to comprehensively demonstrate their therapeutic applications. Synthetic gene circuits can be customized for precise control of therapeutic interventions, leading to personalized therapies that respond specifically to individual patient needs, enhancing treatment efficacy and minimizing side effects. They can be highly sensitive biosensors that provide real-time therapy by accurate monitoring various biomarkers or pathogens and appropriately synthesizing a therapeutic molecule. Synthetic gene circuits may also lead to the development of advanced regenerative therapies and to implantable biodevices that produce on-demand bioactive molecules. However, this technology faces challenges for commercial profitability. The genetic circuit designs need adjustments for specific applications, and may have disadvantages like toxicity from multiple regulators, homologous recombination, context dependency, resource overuse, and environmental variability.Copyright © 2024 Carneiro, Rocha, Damasceno, Barbosa and Soares.