By Kazunari Taira, Kazunori Kataoka, Takuro Niidome
The improvement of gene drugs in keeping with the concept that of molecular remedy has opened new clinical horizons. one of several thoughts in gene drugs, gene layout and supply are particularly major in scientific purposes. This booklet provides cutting-edge info on non-viral gene-delivery suggestions, overlaying a vast spectrum of disciplines that come with chemistry, molecular biology, mobilephone biology, and pharmacokinetics. significant sections introduce molecules for gene supply and their houses; applied sciences of managed gene supply in vitro and in vivo; healing genes and the prestige of scientific functions; and the layout of genes in keeping with present RNA expertise, with innovative advancements on the planet of RNAi proven to be key components in gene medication. This groundbreaking paintings is a useful source for researchers and engineers in genetic engineering, molecular medication, biochemical engineering, and biotechnology.
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Extra info for Non-viral gene therapy: gene design and delivery
2). Other polycations, such as chitosan (Lee et al. 1998), polyethyleneimine (Godbey et al. 1999b; Pollard et al. 1998; Demeneix et al. 1998), and cationic polymethacrylate derivatives (van de Wetering et al. 1998; H2N H N n NH2 N N n N NH2 CH2 CH m N CH CH2 N(CH3)2 CH3 N O NH 2 NH 2 NH2 NH2 NH2 NH2 NH NH 2 N NH 2 N H 2 O NH 2 2 NH2 NH2 2 NH 2 NH NH 2 R1 : -CH3 or -H R2 : -O- or -NHR3 : -CH2CH2-N-(CH2-CH3)2, -CH2CH2-N-(CH3)2 etc. N H N 2 NH2 Cationic vinyl polymers O NH NH 2 12-20 % of R : -H 88-80% of R : -(CH2)2-N-(CH2-CH3)2 NH2 NH n O R3 n NH 2 2 NH 2 OR n 2 NH NH 2 NH R2 DEAE-dextran 3 CH3 2 2 NH NH NH 2 CH2 OR 2Br (CH2) Polybrene (PB) 2 O CH3 (CH3)6 N CH3 R, OR Poly(L-arginine) n Poly(vinyl pyrollidone) CH2 C O NH C Poly(L-lysine) (PLL) NH2 PVA3 n H (CH2)4 2 O CH C NH2 CH2 CH CH2 CH O n NH2 NH2 n OH CH C H2N Branched polyethyleneimine (b-PEI) CH2 CH N H (CH2)4 n n N Linear polyethyleneimine (l-PEI) O O n NH N H N N NH H2N Polyamidoamine dendrimer (PAMAM) Fig.
Maruyama Fig. 1. Schematic illustration of the immobilization of antibody on liposomes. Type A: PEG-free immunoliposomes with antibody covalently linked to the short anchor N-glutaryl-phosphatidylethanolamine (NGPE); type B: PEG-immunoliposomes with antibody covalently linked to NGPE; type C: PEG-immunoliposomes with antibody attached to the distal terminal of distearoyl-N-(3-carboxypropionoyl poly(ethylene glycol) succinyl) phosphatidylethanolamine (DSPE-PEG-COOH), -maleimide or -hydorazide, so-called pendant-type PEG-immunoliposomes A major development in the last few years has been the synthesis of liposomes with a prolonged circulation time in blood, commonly called long-circulating or sterically stabilized liposomes.
J Biol Chem 269:2550–2561 Felgner PL, Gadek TR, Holm M, Roman R, Chan HW, Wenz M, Northrop JP, Ringold GM, Danielsen M (1987) Lipofection: a highly efﬁcient lipid-mediated DNA trancefection procedure. Proc. Natl Acid Sci USA 84:7413–7417 Fenske D, MacLachlan I, Cullis PR (2002) Stabilized plasmid-lipid particles: A systemic gene therapy vector method in engymology 346 ed by M I Phillips 36–71 Gabizon A (1992) Cancer Res 52:891–896 Gabizon A, Papahadjopoulos D (1988) Liposome formulations with prolonged circulation time in blood and enhanced uptake by tumors.