13044-37-6Relevant academic research and scientific papers
Lipid nanoparticle formulations of non-viral capsid-free DNA vectors
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Paragraph 0403; 0408, (2020/09/26)
Provided herein are lipid nanoparticle formulations that comprise an ionizable lipid and non-viral, capsid-free DNA vectors with covalently-closed ends.
LINOLEIC ACID DERIVATIVES, PHARMACEUTICAL COMPOSITION OR FOOD COMPOSITION COMPRISING SAID LINOLEIC ACID DERIVATIVES, AND THEIR USES
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, (2019/12/16)
This invention relates to linoleic acid derivative of Formula (I) below comprising a hydrophobic part C17H31 linked to a polar head part "A": wherein said polar head part A is selected from A1 to A4 below: wherein R1 and R2 are independently selected from the group composed of H or a saturated or unsaturated, straight or branched alkyl group containing 1 to 8 carbon atoms, or R1 and R2 are linked together to form a divalent radical of formula -R1-R2-, wherein -R1-R2- is preferably -CH2-CH2- or-(CH2)3-; R3 is independently selected from the group composed of:
3 - (1 - Piperazine - 4 - yl uncle ding yangtang acid radical) propionic acid amphiphilic derivatives and use thereof (by machine translation)
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Paragraph 0052; 0070; 0078; 0079; 0080, (2018/07/30)
The invention relates to an amphiphilic derivative of 3-(1-tert-butoxycarbonylpiperazin-4-yl)propionic acid, and a use thereof, and also relates to a liposome prepared from the amphiphilic derivative. The use is the use of the liposome as a drug carrier delivery system. The liposome prepared from the amphiphilic derivative can be compounded with gene drug siRNA to form a compound with small particle size and uniform particle size distribution. The amphiphilic derivative is electrically neutral in environment with the pH value of 7.4, increases the in vivo stability of the lipid compound and reduces cytotoxicity caused by too many positive charges. The liposome can specifically inhibit gene expression in human non-small cell lung cancer H1299-Pg13 cells in vitro, and can specifically transship fluorescence gene drugs into normal mouse liver cells in vivo.
COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS
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Paragraph 00725, (2019/01/08)
The disclosure features amino lipids and compositions involving the same. Nanoparticle compositions include an amino lipid as well as additional lipids such as phospholipids, structural lipids, PEG lipids, or a combination thereof. Nanoparticle compositions further including therapeutic and/or prophylactic agents such as RNA are useful in the delivery of therapeutic and/or prophylactic agents to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
Di-aliphatic substituted pegylated lipids
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Page/Page column 103, (2017/08/08)
The disclosure relates to di-aliphatic substituted PEGylated lipids and to methods of their preparation. The disclosure also relates to lipid conjugates containing di-aliphatic substituted PEGylated lipids, and to the use of di-aliphatic substituted PEGylated-lipid conjugates in drug delivery.
COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS
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Page/Page column 00836; 00657; 00868, (2017/07/14)
The disclosure features amino lipids and compositions involving the same. Nanoparticle compositions include an amino lipid as well as additional lipids such as phospholipids, structural lipids, PEG lipids, or a combination thereof. Nanoparticle compositions further including therapeutic and/or prophylactic agents such as RNA are useful in the delivery of therapeutic and/or prophylactic agents to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
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Paragraph 001406, (2017/04/23)
Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
LIPID MEMBRANE STRUCTURE FOR siRNA INTRACELLULAR DELIVERY
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Paragraph 0092-0094, (2017/10/10)
A lipid membrane structure encapsulating an siRNA inside thereof and containing a lipid compound of the formula (I) as a lipid component (R1 and R2 represent CH3—(CH2)n—CH═CH—CH2—CH═CH—(CH2)m—, n represents an integer of 3 to 5, m represents an integer of 6 to 10, p represents an integer of 2 to 7, and R3 and R4 represent a C1-4 alkyl group or a C2-4 alkenyl group.
OLIGONUCLEOTIDES, COMPOSITIONS AND METHODS THEREOF
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Paragraph 001647, (2018/01/17)
The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.
3 - ((2 - (Dimethylamino) ethyl) (methyl) amino) propionic acid amphiphilic derivatives and use thereof
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Paragraph 0053; 0071-0074; 0081-0083, (2018/01/05)
The invention relates to an amphiphilic derivative of 3-((2-dimethylamino)ethyl(methyl)amino)propionic acid, and a use thereof, and also relates to a liposome prepared from the above amphiphilic compound. The use is the use of the liposome as a drug carrier delivery system. The liposome prepared from the amphiphilic compound has many positive charges in acidic environment, and can be well compounded with gene drug siRNA to form a compound with small particle size and uniform particle size distribution. The amphiphilic compound is electrically neutral in environment with the pH value of 7.4, increases the in vivo stability of the lipid compound and reduces cytotoxicity caused by too many positive charges. The liposome can specifically inhibit gene expression in human non-small cell lung cancer H1299-Pg13 cells in vitro, and can specifically transship fluorescence gene drugs into normal mouse liver cells in vivo.
