David et al: Current status and future direction of fetal gene therapy Gerdts V, Babiuk LA, van Drunen Littel-van den Hurk, Griebel PJ (2000) Fetal immunization by a DNA vaccine delivered into 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 immunity and immune memory in the neonate. J Immunol 168, 18771885. 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 and persistent gene expression in heart and respiratory and limb musculature of mice demonstrate applicability of EIAV lentivirus for gene therapy of Duchenne/Becker Muscular Dystrophies. Hum Gene Ther submitted. Gregory LG, Harbottle RP, Lawrence L, Knapton HJ, Themis M, Coutelle C (2002) Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo. Mol Ther 7, 1-8. Haake AR, Cooklis M (1997) Incomplete differentiation of fetal keratinocytes in the skin equivalent leads to the default pathway 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-linked severe 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 diaphragmatic hernia in utero IX: fetuses with poor prognosis (liver herniation and low lung-to-head ratio) can be saved by fetoscopic temporary tracheal occlusion. J Pediatr Surg 33, 1017-1022. Hatzoglou M, Lamers W, Bosch F, Wynshaw-Boris A, Clapp DW, Hanson RW (1990) Hepatic gene transfer in animals using retrovirus containing the promoter from the gene for phosphoenolpyruvate carboxykinase. J Biol Chem 265, 17285-17293. Hatzoglou M, Moorman A, Lamers W (1995) Persistent expression of genes transferred in the fetal rat liver via retroviruses. Somat Cell Mol Genet 21, 265-278. Heikkil채 A, Hiltunen MO, Turunen MP, Keski-Nisula L, Turunen A-M, R채s채nen H, Rissanen TT, Kosma V-M, Manninen H, Heinonen S, Yl채-Herttuala S (2001) Angiographically guided utero-placental gene transfer in rabbits with adenoviruses, plasmid/liposomes and plasmid/polyethyleneimine complexes. Gene Ther 8, 784788. Hendrickx AG, Peterson PE (1997) Perspectives on the use of the baboon in embryology and teratology research. Human Reprod Update 3, 575-592. Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous DM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA (1999) Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adenoassociated viral vector. Nat Med 5, 56-63. Holzgreve W, Golbus MS (1986) Prenatal diagnosis of ornithine transcarbamylase deficiency utilizing fetal liver biopsy. Am JHuman Genet 36, 320-328. Holzinger A, Trapnell BC, Weaver TE, Whitsett JA, Iwamoto HS (1995) Intraamniotic administration of an adenoviral vector for gene transfer to fetal sheep and mouse tissues. Ped Res 38, 844-850.
Horn HM, Tidman MJ (2002) The clinical spectrum of dystrophic epidermolysis bullosa. Br J Dermatol 146, 267274. Hubeau C, Puchelle E, Gaillard D (2001) Distinct pattern of immune cell population in the lung of human fetuses with cystic 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-like particle formation by F-deficient Sendai virus is temperature sensitive and reduced by mutations in M and HN proteins. J Virol 77, 3238-3246. Iwamoto HS, Trapnell BC, McConnell CJ, Daugherty C, Whitsett JA ( 1999) Pulmonary inflammation associated with repeated, prenatal exposure to an E1, E3-deleted adenoviral vector in sheep. Gene Ther 6, 98-106. Jauniaux E, Gulbis B (2000) Fluid compartments of the embryonic environment. Hum Reprod Update 6, 268-278. Jauniaux E, Gulbis B, Gerloo E (1999) Free amino acids in human fetal liver and fluids at 12 - 17 weeks of gestation. Hum Reprod 14, 1638-1641. Jesudason EC (2002) Challenging embryological theories on congenital diaphragmatic hernia: future therapeutic implications for paediatric surgery. Ann R Coll Surg Engl 84, 252-259. Jeyakumar M, Butters TD, Dwek RA, Platt FM (2002) Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis. Neuropathol Appl Neurobiol 28, 343-357. Johnson LG, Olsen JC, Sarkadi B, Moore KL, Swanstrom R, Boucher RC (1992) Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 2, 21-25. Johnson LG, Vanhook MK, Coyne CB, Haykal-Coates N, Gavett SH (2003) Safety and efficiency of modulating paracellular permeability to enhance airway epithelial gene transfer in vivo. Hum Gene Ther 14, 729-747. Johnston J, Tazelaar J, Rivera VM, Clackson T, Gao GP, Wilson JM (2003) Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model. Mol Ther 7, 493-497. Juengst ET (2003) What next for human gene therapy? BMJ 326, 1410-1411. Kalache KD, Chaoui R, Marcks B, Nguyen-Dobinsky TN, Wernicke KD, Wauer R, Bollmann R (2000) Differentiation between human fetal breathing patterns by investigation of breathing-related tracheal fluid flow velocity using Doppler sonography. 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) Longterm normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII. Gene 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 hepatic gene therapy: Complete albeit transient correction of factor IX 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 expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24, 257-261. Kay MA, Rothenberg SR, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC (1993) In vivo gene therapy of hemophilia B:
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