Expression of exogenous polynucleotide sequences in a vertebrate
申请人:VICAL, INC.
公开号:EP1026253A2
公开(公告)日:2000-08-09
A method for delivering an isolated polynucleotide to the interior of a cell in a vertebrate, comprising the interstitial introduction of an isolated polynucleotide into a tissue of the vertebrate where the polynucleotide is taken up by the cells of the tissue and exerts a therapeutic effect on the vertebrate. The method can be used to deliver a therapeutic polypeptide to the cells of the vertebrate, to provide an immune response upon in vivo translation of the polynucleotide, to deliver antisense polynucleotides, to deliver receptors to the cells of the vertebrate, or to provide transitory gene therapy.
Method for coating fine particle with lipid membrane
申请人:Kyowa Hakko Kirin Co., Ltd.
公开号:EP2241308A2
公开(公告)日:2010-10-20
An object of the present invention is to provide a safe, convenient and efficient method for coating fine particles with lipid membrane.
There is provided a method for coating fine particles with lipid membrane, characterized in that, the rate of a polar organic solvent in an aqueous solution containing the polar organic solvent where the fine particles are dispersed and lipid is dissolved, is decreased.
The present invention provides a composition for suppressing the expression of the CKAP5 gene, the composition comprising a lipid particle containing a double-stranded nucleic acid as a drug and a cationic lipid,
the double-stranded nucleic acid having an antisense strand having a base sequence complementary to the sequence of at least 19 continuous bases in CKAP5 gene mRNA of any one of SEQ ID NOs: 1 to 6,
the cationic lipid being represented by formula (I):
wherein
R1 and R2 are the same or different and are each linear or branched alkyl, alkenyl or alkynyl of 12 to 24 carbon atoms;
L1 and L2 are the same or different and are each -CO-O- or -O-CO-;
a and b are the same or different and are each 1 to 3; and
R3 is a hydrogen atom, alkyl of 1 to 6 carbon atoms, or alkenyl of 3 to 6 carbon atoms;
a medicament comprising the composition, and the like.
What is described is a method of producing lipid nanoparticles having an average diameter of less than 100 nm, comprising pumping an aqueous buffer solution through a 1st tube by a 1st HPLC pump, pumping an organic lipid solution through a 2nd tube by a 2nd HPLC pump, wherein the 2nd tube intersects the 1st tube perpendicularly within a mixing module, and wherein the organic lipid solution is mixed with the aqueous solution in a turbulent flow within the mixing module.
COMPOSITION FOR INCREASING EXPRESSION OF BLOOD COAGULATION FACTOR GENE, COMPRISING CORE-SHELL STRUCTURED MICROPARTICLES AS ACTIVE INGREDIENT
申请人:G&P Bioscience Co., Ltd.
公开号:EP3753550A1
公开(公告)日:2020-12-23
The present disclosure relates to a composition for increasing the expression of a blood coagulation factor gene, which contains core-shell structured microparticles as an active ingredient. When administered in vivo along with blood coagulation factor VIII gene or a variant gene thereof, the composition for increasing the expression of a blood coagulation factor gene of the present disclosure can increase the expression of the gene by at least 30%. When administered along with a gene therapeutic agent, the composition can achieve a therapeutic effect even with a very small amount of a gene, and thus is useful.
本公开涉及一种用于提高凝血因子基因表达的组合物,该组合物含有核壳结构微粒作为活性成分。当与血凝因子 VIII 基因或其变异基因一起在体内给药时,本公开的用于增加血凝因子基因表达的组合物可使该基因的表达量增加至少 30%。当与基因治疗剂一起给药时,该组合物即使使用极少量的基因也能达到治疗效果,因此非常有用。