📜1-溴-顺-9-十八碳烯,3-二甲胺基-1,2-丙二醇置于sodium Hydride体系中,用 苯 用作溶剂,化学反应 18.5H,以65%的收率获得1,2-二油醇-3-二甲基氨基-丙烷(dodma) 参考文献:A Scalable,Extrusion-Free Method For Efficient Liposomal Encapsulation Of Plasmid Dna 标题:A Scalable,Extrusion-Free Method For Efficient Liposomal Encapsulation Of Plasmid Dna 摘要:开发了一种完全可扩展且无挤出步骤的方法,用于快速,可重复地制备稳定质粒脂质颗粒(splp),用于非病毒的全身基因治疗.通过以受控,逐步的方式,将溶解在乙醇中的脂质与含dna的水溶液混合,瞬时形成包裹质粒dna的脂质体.将dna缓冲液和脂质乙醇流在t形混合室中结合,导致乙醇瞬时稀释至支持脂质溶解所需的浓度以下.所得到的含dna脂质体通过第二步逐步稀释进一步稳定.使用这种方法,制备了粒径小于200 Nm,Dna包封效率大于80%的单分散颗粒.在小鼠neuro 2A肿瘤模型中,Splp的体内循环半衰期为13小时,非常好的肿瘤积聚和基因表达谱与之前通过洗涤剂透析制备的splp相似.冷冻传输电子显微镜分析显示,通过逐步乙醇稀释制备的splp是由单层,双层和多层囊泡混合组成的.同样脂质组成,不含dna的囊泡粒径也小于200 Nm,但主要是双层,形态异常延长,表明质粒颗粒影响了包裹脂质体的形态.类似方法还用于制备中性蛋黄磷脂酰胆碱:胆固醇(epc:Chol)脂质体,具有ph梯度,通过脂溶性阳离子碱性藏红o的摄取得以证实.这种方法将实现splp的扩大生产和制造,满足临床前和临床研究的需求.此外,这种方法现在允许加速splp配方的开发,使新型载体的快速开发和评估成为可能. Doi:10.1007/s11095-004-1873-Z
专利信息
专利号:US-5455157-A 优先权日:1989-05-22 标 题:Method for the non-radioactive measurement of the nucleic acid synthesis in eukaryotic cells 发明人:HINZPETER MATTHIAS; ELTZ HERBERT VON DER 权利人:BOEHRINGER MANNHEIM GMBH 摘要:For the determination of the rate of nucleic acid synthesis in eukaryotic cells a nucleotide which is non-radioactively labelled or derivatized with a hapten is introduced into the cells with the addition of liposomes and the rate of synthesis of the nucleic acids is determined by means of the incorporation of the nucleotide via its label or derivatization.
专利号:US-2025049714-A1 优先权日:2021-12-09 标题 :Synthesis of ester, carbonate, and carbamate-derived novel biodegradable ionizable lipids from methyl ricinoleate or methyl 12-hydroxystearate and its applications 发明人:ANDERSON DANIEL GRIFFITH; RHYM LUKE HYUNSIK; LI BOWEN; GORDON AKIVA; RAJITH SINGH MANAN; WITTEN JACOB 权利人:MASSACHUSETTS INST TECHNOLOGY 摘要:Provided herein are compounds, such as compounds of Formula (I), and pharmaceutically acceptable salts, hydrates, solvates, polymorphs, co-crystals, tautomers, stereoisomers, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of polynucleotides, such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, liver disease, spleen disease, lung disease, painful condition, psychiatric disorder, musculoskeletal disease, a metabolic disorder, inflammatory disease, or autoimmune disease).
专利号:US-11560562-B2 优先权日:2017-02-27 标题 :Large-scale synthesis of messenger RNA 发明人:DIAS ANUSHA; CRAWFORD DANIEL; DEROSA FRANK; ABYSALH JONATHAN; HEARTLEIN MICHAEL 权利人:TRANSLATE BIO INC 摘要:The present invention provides methods for large-scale production of a composition enriched for full-length mRNA molecules using an SP6 RNA polymerase and compositions produced using such methods and uses thereof.
专利号:US-2022136022-A1 优先权日:2020-10-15 标 题 :Large Scale Synthesis of Messenger RNA 发明人:ABYSALH JONATHAN; DIAS ANUSHA; VARGAS JOREL E; COOPER DUSTIN; DEROSA FRANK 权利人:TRANSLATE BIO INC 摘要:The present invention provides, among other things, methods for large-scale production of a composition comprising full-length messenger RNA product that is substantially free of double-stranded RNA, and compositions produced using such methods and uses thereof. The present invention is based, in part, on the surprising discovery that mRNA product produced by in vitro transcription using an SP6 RNA polymerase is substantially free of double-stranded RNA. In one aspect, the present invention provides methods of generating large-scale mRNA product for mRNA therapy without need for a chromatography step.
专利号:US-9790531-B2 优先权日:2012-08-14 标 题 :Enzymes and polymerases for the synthesis of RNA 发明人:WANG YUXUN; RAMAKRISHNAN DIVAKAR; DE FOUGEROLLES ANTONIN; WHORISKEY SUSAN 权利人:MODERNATX INC 摘要:The invention relates to compositions and methods for the design, evolution, preparation, and/or manufacture of enzymes for use with polynucleotides, primary transcripts and mmRNA molecules.
专利号:US-2018312534-A1 优先权日:2015-10-16 标题:Fluorescent anticancer platinum drugs 发明人:SARKAR ARINDAM; MANDAL SWADHIN KUMAR; SENGUPTA ANIRUDDHA; BISWAS GOUTAM; DUTTA PRADIP; SHARMA RUPALI; RAJ JUSTIN PAUL; SURYAVANSHI HEMANT; KUMARI SMITA 权利人:INVICTUS ONCOLOGY PVT LTD 摘要:The present disclosure is in relation to the field of nanotechnology and cancer therapeutics. In particular, the present disclosure relates to fluorescent platinum based compounds. The disclosure further relates to synthesis of said fluorescent platinum based compounds, nanoparticles and compositions comprising said fluorescent platinum based compounds/nanoparticles. The disclosure also relates to methods of managing cancer by the fluorescence changes between aforesaid platinum based compounds and corresponding free ligands, nanoparticles and compositions.