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GTMB 7 - Gene Therapy & Molecular Biology

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David et al: Current status and future direction of fetal gene therapyGerdts V, Babiuk LA, van Drunen Littel-van den Hurk, GriebelPJ (2000) Fetal immunization by a DNA vaccine deliveredinto the oral cavity. Nat Med 6, 929-932.Gerdts V, Snider M, Brownlie R, Babiuk LA, Griebel PJ (2003)Oral DNA vaccination in utero induces mucosal immunityand immune memory in the neonate. J Immunol 168, 1877-1885.Gregory L, Waddington SN, Holder M, Mitrophanous K,Buckley SMK, Bigger B, Ellard FM, Walmsley LE,Lawrence L, Cook T, Al-Allaf F, Kingsman S, Coutelle C,Themis M (2003) Highly efficient in utero gene transfer andpersistent gene expression in heart and respiratory and limbmusculature of mice demonstrate applicability of EIAVlentivirus for gene therapy of Duchenne/Becker MuscularDystrophies. Hum <strong>Gene</strong> Ther submitted.Gregory LG, Harbottle RP, Lawrence L, Knapton HJ, Themis M,Coutelle C (2002) Enhancement of adenovirus-mediatedgene transfer to the airways by DEAE dextran and sodiumcaprate in vivo. Mol Ther 7, 1-8.Haake AR, Cooklis M (1997) Incomplete differentiation of fetalkeratinocytes in the skin equivalent leads to the defaultpathway of apoptosis. Exp Cell Res 231, 83-95.Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C,De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R,Dupuis-Girod S, Fischer A, Davies EG, Kuis W, Leiva L,Cavazzana-Calvo M (2002) Sustained correction of X-linkedsevere combined immunodeficiency by ex vivo gene therapy.N Engl J Med 346, 1185-1193.Harrison MR, Mychaliska GB, Albanese CT, Jennings RW,Farrell JA, Hawgood S, Sandberg P, Levine AH, Lobo E,Filly RA (1998) Correction of congenital diaphragmatichernia in utero IX: fetuses with poor prognosis (liverherniation and low lung-to-head ratio) can be saved byfetoscopic temporary tracheal occlusion. J Pediatr Surg 33,1017-1022.Hatzoglou M, Lamers W, Bosch F, Wynshaw-Boris A, ClappDW, Hanson RW (1990) Hepatic gene transfer in animalsusing retrovirus containing the promoter from the gene forphosphoenolpyruvate carboxykinase. J Biol Chem 265,17285-17293.Hatzoglou M, Moorman A, Lamers W (1995) Persistentexpression of genes transferred in the fetal rat liver viaretroviruses. Somat Cell Mol <strong>Gene</strong>t 21, 265-278.Heikkilä A, Hiltunen MO, Turunen MP, Keski-Nisula L,Turunen A-M, Räsänen H, Rissanen TT, Kosma V-M,Manninen H, Heinonen S, Ylä-Herttuala S (2001)Angiographically guided utero-placental gene transfer inrabbits with adenoviruses, plasmid/liposomes andplasmid/polyethyleneimine complexes. <strong>Gene</strong> Ther 8, 784-788.Hendrickx AG, Peterson PE (1997) Perspectives on the use ofthe baboon in embryology and teratology research. HumanReprod Update 3, 575-592.Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D,Fields PA, Burton M, Bellinger DA, Read MS, BrinkhousDM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA(1999) Long-term correction of canine hemophilia B by genetransfer of blood coagulation factor IX mediated by adenoassociatedviral vector. Nat Med 5, 56-63.Holzgreve W, Golbus MS (1986) Prenatal diagnosis of ornithinetranscarbamylase deficiency utilizing fetal liver biopsy. AmJHuman <strong>Gene</strong>t 36, 320-328.Holzinger A, Trapnell BC, Weaver TE, Whitsett JA, IwamotoHS (1995) Intraamniotic administration of an adenoviralvector for gene transfer to fetal sheep and mouse tissues. PedRes 38, 844-850.Horn HM, Tidman MJ (2002) The clinical spectrum ofdystrophic epidermolysis bullosa. Br J Dermatol 146, 267-274.Hubeau C, Puchelle E, Gaillard D (2001) Distinct pattern ofimmune cell population in the lung of human fetuses withcystic fibrosis. J Allergy Clin Immunol 108, 524-529.Inoue M, Tokusumi Y, Ban H, Kanaya T, Tokusumi T, Nagai Y,Iida A, Hawegawa M (2003) Nontransmissible virus-likeparticle formation by F-deficient Sendai virus is temperaturesensitive and reduced by mutations in M and HN proteins. JVirol 77, 3238-3246.Iwamoto HS, Trapnell BC, McConnell CJ, Daugherty C,Whitsett JA (1999) Pulmonary inflammation associated withrepeated, prenatal exposure to an E1, E3-deleted adenoviralvector in sheep. <strong>Gene</strong> Ther 6, 98-106.Jauniaux E, Gulbis B (2000) Fluid compartments of theembryonic environment. Hum Reprod Update 6, 268-278.Jauniaux E, Gulbis B, Gerloo E (1999) Free amino acids inhuman fetal liver and fluids at 12 - 17 weeks of gestation.Hum Reprod 14, 1638-1641.Jesudason EC (2002) Challenging embryological theories oncongenital diaphragmatic hernia: future therapeuticimplications for paediatric surgery. Ann R Coll Surg Engl84, 252-259.Jeyakumar M, Butters TD, Dwek RA, Platt FM (2002)Glycosphingolipid lysosomal storage diseases: therapy andpathogenesis. Neuropathol Appl Neurobiol 28, 343-357.Johnson LG, Olsen JC, Sarkadi B, Moore KL, Swanstrom R,Boucher RC (1992) Efficiency of gene transfer forrestoration of normal airway epithelial function in cysticfibrosis. Nat <strong>Gene</strong>t 2, 21-25.Johnson LG, Vanhook MK, Coyne CB, Haykal-Coates N, GavettSH (2003) Safety and efficiency of modulating paracellularpermeability to enhance airway epithelial gene transfer invivo. Hum <strong>Gene</strong> Ther 14, 729-747.Johnston J, Tazelaar J, Rivera VM, Clackson T, Gao GP, WilsonJM (2003) Regulated expression of erythropoietin from anAAV vector safely improves the anemia of beta-thalassemiain a mouse model. Mol Ther 7, 493-497.Juengst ET (2003) What next for human gene therapy? BMJ326, 1410-1411.Kalache KD, Chaoui R, Marcks B, Nguyen-Dobinsky TN,Wernicke KD, Wauer R, Bollmann R (2000) Differentiationbetween human fetal breathing patterns by investigation ofbreathing-related tracheal fluid flow velocity using Dopplersonography. Prenat Diagn 20, 45-50.Kamata Y, Tanabe A, Kanaji A, Kosuga M, Fukuhara Y, Li XK,Suzuki S, Yamada M, Azuma N, Okuyama T (2003) Longtermnormalization in the central nervous system, ocularmanifestations, and skeletal deformities by a single systemicadenovirus injection into neonatal mice withmucopolysaccharidosis VII. <strong>Gene</strong> Ther 10, 406-414.Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F,Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR,Read M, Brinkhous KM, Woo SLC (1994) In vivo hepaticgene therapy: Complete albeit transient correction of factorIX deficiency in hemophilia B dogs. Proc Natl Acad Sci 91,2353-2357.Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB,McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW,Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW,High KA (2000) Evidence for gene transfer and expressionof factor IX in haemophilia B patients treated with an AAVvector. 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