体外嵌合抗原受体T细胞(ex vivo CAR-T)在改善血液系统恶性肿瘤(尤其是B细胞恶性肿瘤)方面显示出卓越的治疗潜力,但其广泛应用面临巨大挑战,包括体外制造工艺复杂、生产成本高昂等因素。近年来,随着RNA药物、靶向递送系统等领域的快速发...体外嵌合抗原受体T细胞(ex vivo CAR-T)在改善血液系统恶性肿瘤(尤其是B细胞恶性肿瘤)方面显示出卓越的治疗潜力,但其广泛应用面临巨大挑战,包括体外制造工艺复杂、生产成本高昂等因素。近年来,随着RNA药物、靶向递送系统等领域的快速发展,体内嵌合抗原受体T细胞(in vivo CAR-T)作为一种创新策略应运而生。in vivo CAR-T通过病毒载体或脂质纳米颗粒(LNPs)等靶向递送系统,将编码CAR的遗传物质直接导入患者体内,实现体内T细胞工程化改造,这一策略从根本上省去了繁琐的体外细胞操作步骤和传统的化疗预处理环节。本研究系统梳理了in vivo CAR-T的技术进展与非临床研究考虑。in vivo CAR-T兼具基因治疗与细胞治疗的双重属性,涉及多种递送载体,类型多样,机制复杂,其非临床研究可遵循基于风险、个案处理的原则,在现有相关技术指导原则框架下,合理设计并开展非临床研究,以获取科学规范的试验数据来支持开展临床试验和批准上市。展开更多
Aim An industrial enzyme β-glucanase was used to transfortn notoginsenoside Fe for the first time. Methods Notoginsenoside Fe was isolated from the leave saponin of Panax notoginseng （Burk.） Chen FH. The enzymatica...Aim An industrial enzyme β-glucanase was used to transfortn notoginsenoside Fe for the first time. Methods Notoginsenoside Fe was isolated from the leave saponin of Panax notoginseng （Burk.） Chen FH. The enzymatically transformed compounds were detected by HPLC and two transformed compounds were identified as 20 （S） -protopanaxadiol-20- O- α-L-arabinofuranosyl （ 1→6 ） - β-gluco- pyranoside, ginsenoside-Mc） and 20（S）-protopanaxadiol-20-O-β-D-glucopyranoside compound-K （C-K） respectively on the basis of their ^1H NMR and ^13 C NMR spectral data. Results Based on the enzymolytic kinetic curve, the transformation rate of notoginsenoside Fe reached 95% after 24 h. Conclusion The enzymatic transformation pathway of notoginsenoside Fe by β-glucanase has been proposed as notoginsenoside Fe→ginsenoside Mc→C-K.展开更多
文摘体外嵌合抗原受体T细胞(ex vivo CAR-T)在改善血液系统恶性肿瘤(尤其是B细胞恶性肿瘤)方面显示出卓越的治疗潜力,但其广泛应用面临巨大挑战,包括体外制造工艺复杂、生产成本高昂等因素。近年来,随着RNA药物、靶向递送系统等领域的快速发展,体内嵌合抗原受体T细胞(in vivo CAR-T)作为一种创新策略应运而生。in vivo CAR-T通过病毒载体或脂质纳米颗粒(LNPs)等靶向递送系统,将编码CAR的遗传物质直接导入患者体内,实现体内T细胞工程化改造,这一策略从根本上省去了繁琐的体外细胞操作步骤和传统的化疗预处理环节。本研究系统梳理了in vivo CAR-T的技术进展与非临床研究考虑。in vivo CAR-T兼具基因治疗与细胞治疗的双重属性,涉及多种递送载体,类型多样,机制复杂,其非临床研究可遵循基于风险、个案处理的原则,在现有相关技术指导原则框架下,合理设计并开展非临床研究,以获取科学规范的试验数据来支持开展临床试验和批准上市。
文摘Aim An industrial enzyme β-glucanase was used to transfortn notoginsenoside Fe for the first time. Methods Notoginsenoside Fe was isolated from the leave saponin of Panax notoginseng （Burk.） Chen FH. The enzymatically transformed compounds were detected by HPLC and two transformed compounds were identified as 20 （S） -protopanaxadiol-20- O- α-L-arabinofuranosyl （ 1→6 ） - β-gluco- pyranoside, ginsenoside-Mc） and 20（S）-protopanaxadiol-20-O-β-D-glucopyranoside compound-K （C-K） respectively on the basis of their ^1H NMR and ^13 C NMR spectral data. Results Based on the enzymolytic kinetic curve, the transformation rate of notoginsenoside Fe reached 95% after 24 h. Conclusion The enzymatic transformation pathway of notoginsenoside Fe by β-glucanase has been proposed as notoginsenoside Fe→ginsenoside Mc→C-K.
