专利号:US-2025049961-A1 优先权日:2021-12-23 标题 :Scalable and high-purity cell-free synthesis of closed-ended dna vectors 发明人:MONDS RUSSELL; CIPI JORIS; BLACKSTOCK DANIEL JASON; DURANT JOHN CHESTER 权利人:GENERATION BIO CO 摘要:This disclosure provides methods for scalable and high-purity cell-free synthesis of DNA vectors, particularly closed-ended DNA vectors (e.g., ceDNA vectors) having linear and continuous structure for delivery and expression of a transgene. The cell-free synthesis includes digesting a double-stranded DNA construct with at least one restriction endonuclease that is capable of cleaving the construct at cleavage sites, which are distinct from the recognition sites, to release an insert having unique overhangs that regulate the high specificity of the subsequent ligation reaction. The insert is then ligated with inverted terminal repeat (ITR) oligonucleotides to form the closed-ended DNA vector. Corresponding DNA vectors prepared by these methods and related products as well other base and intermediate vectors and constructs associated with the methods are also provided in this disclosure.
专利号:US-9365607-B1 优先权日:2012-07-31 标题 :Synthesis of deuterated ribo nucleosides, N-protected phosphoramidites, and oligonucleotides 发明人:SRIVASTAVA SURESH C; YASKO AMY 权利人:ASED LLC 摘要:The present invention is directed towards the synthesis of high purity deuterated sugars, deuterated phosphoramidites, deuterated nucleobases, deuterated nucleosides, deuterated oligonucleotides, and deuterated RNA's of defined sequences which can exhibit biochemically useful and biologically valuable properties, thus having potential for therapeutic uses.
专利号:US-12442015-B2 优先权日:2018-01-19 标 题:Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors 发明人:ALKAN OZAN; KOTIN ROBERT MICHAEL; STANTON MATTHEW; KERR DOUGLAS ANTHONY; PELLETIER CAROLYN 权利人:GENERATION BIO CO 摘要:The application describes methods for synthetic synthesis and cell-free synthesis of DNA vectors, particularly closed-ended DNA vectors (e.g., ceDNA vectors) having linear and continuous structure for delivery and expression of a transgene. The present invention relates to an in vitro process for production of closed-ended DNA vectors, corresponding DNA vector products produced by the methods and uses thereof, and oligonucleotides and kits useful in the process of the invention. DNA vectors produced using the methods described herein are free from unwanted side effects due to contaminants introduced during production in cell lines, for example, bacterial or insect cell lines. Further provided herein are methods and cell lines for reliable gene expression in vitro, ex vivo and in vivo using the ceDNA vectors synthesized using the methods herein.
专利号:US-7956169-B1 优先权日:2010-06-16 标题 :Synthesis of novel azo-dyes and their use in oligonucleotide synthesis 发明人:LAIKHTER ANDREI; SRIVASTAVA SURESH C; SRIVASTAVA NAVEEN P 权利人:CHEMGENES CORP 摘要:The invention provides a novel group of azo quencher compositions that are useful as quenchers of fluorescence and to methods for making and using them. The quenchers contain an azo bond and 1,3,3-trimethyl-2-methyleneindoline ring system. The quenchers can be derivatized to facilitate their conjugation to a variety of biologically relevant compounds, including lipids, nucleic acids, peptides, proteins, and the like.
专利号:US-9254327-B2 优先权日:2010-05-10 标 题 :Methods and compositions for delivery of active agents 发明人:MANOHARAN MUTHIAH; RAJEEV KALLANTHOTTATHIL G 权利人:MANOHARAN MUTHIAH; RAJEEV KALLANTHOTTATHIL G; ALNYLAM PHARMACEUTICALS INC 摘要:A lipid particle can include a cationic lipid. Synthesis of the cationic lipid can include a ylide-based reaction, such as a Wittig reaction or sulfur ylide reaction. In some cases, the synthesis can also include a Michael addition or a related addition reaction.
专利号:US-2024123076-A1 优先权日:2021-02-08 标题:Unsaturated dendrimers compositions, related formulations, and methods of use thereof 发明人:LEE SANG M; SIEGWART DANIEL J 权利人:UNIV TEXAS 摘要:Described herein are novel lipid compositions comprising unsaturated dendrimers and methods of synthesis of unsaturated dendrimers. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Also described herein are pharmaceutical formulations comprising an unsaturated dendrimer, a lipid composition, and a therapeutic agent. Further described in here are methods of mRNA delivery comprising a lipid composition and a therapeutic agent. Further described herein are high-potency dosage forms of a therapeutic formulated with a lipid composition.
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