GTMB 7 - Gene Therapy & Molecular Biology

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David et al: Current status and future direction of fetal gene therapymucopolysaccharidosis type VII using nonprimate lentiviralvectors. Mol Ther 3, 850-856.McCray PB, Armstrong K, Zabner J, Miller DW, Koretzky GA,Couture L, Robillard JE, Smith AE, Welsh MJ (1995)Adenoviral-mediated gene transfer to fetal pulmonaryepithelia in vitro and in vivo. J Clin Invest 95, 2620-2632.Meertens L, Zhao Y, Rosic-Kablar S, Li L, Chan K, Dobson H,Gartley C, Lutzko C, Hopwood J, Kohn D, Kruth S, HoughMR, Dube ID (2002) In utero injection of alpha-Liduronidase-carryingretrovirus in caninemucopolysaccharidosis type I: infection of multiple tissuesand neonatal gene expression. Hum Gene Ther 13, 1809-1820.Miller DG, Adam MA, Miller AD (1990) Gene transfer byretrovirus vectors occurs only in cells that are activelyreplicating at the time of infection. Mol Cell Biol 10, 4239-4242.Mitchell M, Jerebtsova M, Batshaw ML, Newman K, Ye X(2000) Long-term gene transfer to mouse fetuses withrecombinant adenovirus and adeno-associated virus (AAV)vectors. Gene Ther 7, 1986-1992.Mitrophanous K, Yoon S, Rohll J, Patil D, Wilkes F, Kim V,Kingsman S, Kingsman A, Mazarakis N (1999) Stable genetransfer to the nervous system using a non-primate lentiviralvector. Gene Ther 6, 1808-1818.Miyagawa Y, Matsuoka T, Baba A, Nakamura T, Tsuno T,Tamura A, Agematsu K, Yabuhara A, Uehara Y, Kawai H(1992) Fetal liver T cell receptor gamma/delta+ T cells ascytotoxic T lymphocytes specific for maternal alloantigens. JExp Med 176, 1-7.Moise KJ, Hesketh DE, Belfort MM, Saade G, Van den VeyerIB, Hudson KM, Rodkey LS (1992) Ultrasound-guidedblood sampling of rabbit fetuses. Lab Anim Sci 42, 398-401.Monahan PE, Samulski RJ (2000) Adeno-associated virusvectors for gene therapy: more pros than cons? Mol MedToday 6, 433-440.Moss IR, Scarpelli EM (1981) Stimulatory effect of theophyllineon regulation of fetal breathing movements. Ped Res 15,870-873.Muench MO, Rai J, Barcena A, Leemhuis T, Farrell J, HumeauL, Maxwell-Wiggins JR, Capper J, Mychaliska GB,Albanese CT, Martin T, Tsukamoto A, Curnutte JT, HarrisonMR (2001) Transplantation of a fetus with paternal Thy-1(+)CD34(+) cells for chronic granulomatous disease. BoneMarrow Transplant 27, 355-364.Murphy SJ, Chong H, Bell S, Diaz RM, Vile RG (2002) Novelintegrating adenoviral/retroviral hybrid vector for genetherapy. Hum Gene Ther 13, 745-760.Nakai H, Montini E, Fuess S, Storm TA, Grompe M, Kay MA(2003) AAV serotype 2 vectors preferentially integrate intoactive genes in mice. Nat Genet 34, 297-302.Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH,Verma IM, Trono D (1996) In vivo gene delivery and stabletransduction of nondividing cells by a lentiviral vector.Science 272, 263-267.Newnham JP, Kelly RW (1993) Ultrasound for research withfetal sheep. In: Neilson JP, Chambers SE (Eds.), ObstetricUltrasound I. Oxford Medical Publications, pp. 203-222.Nicolini U, Nicolaidis P, Fisk NM, Tannirandorn Y, Rodeck C(1990) Fetal blood sampling from the intrahepatic vein:analysis of safety and clinical experience with 214procedures. Obstet Gynaecol 76, 47-53.Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, CalosMP (2002) Site specific genomic integration producestherapeutic Factor IX levels in mice. Nat Biotechnol 20,1124-1128.Olivieri NF (1999) The beta-thalassaemias. N Engl J Med 341,99-109.Olivieri NF, Weatherall DJ (1998) The therapeutic reactivationof fetal haemoglobin. Hum Mol Genet 7, 1655-1658.Ortiz-Urda S, Thyagarajan B, Keene DR, Lin Q, Calos MP,Khavari PA (2003) _C31 Integrase-Mediated NonviralGenetic Correction of Junctional Epidermolysis Bullosa.Hum Gene Ther 14, 923-928.Pahal GS, Jauniaux E, Kinnon C, Thrasher AJ, Rodeck C (2000)Normal development of human fetal hematopoiesis betweeneight and seventeen weeks' gestation. Am J Obstet Gynecol183, 1029-1034.Pai S, Marinkovich MP (2002) Epidermolysis bullosa: new andemerging trends. Am J Clin Dermatol 3, 371-380.Palmer TD, Rosman GJ, Osborne WRA, Miller D (1991)Genetically modified skin fibroblasts persist long aftertransplantation but gradually inactivate introduced genes.Proc Natl Acad Sci 88, 1330-1334.Papadopulos NA, Dumitrascu I, Ordonez JL, Decaluwe H, LerutTE, Barki G, Deprest JA (1999) Fetoscopy in the pregnantrabbit at midgestation. Fetal Diagn Ther 14, 118-121.Pardi G, Marconi AM, Cetin I (2002) Placental-fetalinterrelationship in IUGR fetuses--a review. Placenta 23suppl A, S136-S141.Parsons DW, Grubb BR, Johnson LG, Boucher RC (1998)Enhanced in vivo airway gene transfer via transientmodification of host barrier properties with a surface-activeagent. Hum Gene Ther 9, 2661-2672.Pawliuk R, Westerman KA, Fabry ME, Payen E, Tighe R,Bouhassira EE, Acharya SA, Ellis J, London IM, Eaves CJ,Humphries RK, Beuzard Y, Nagel RL, Leboulch P (2001)Correction of sickle cell disease in transgenic mouse modelsby gene therapy. Science 294, 2368-2371.Peault B, Tirouvanziam R, Sombardier MN, Chen S, PerricaudetM, Gaillard D (1994) Gene transfer to human fetalpulmonary tissue developed in immunodeficient SCID mice.Hum Gene Ther 5, 1131-1137.Peebles D, Gregory LG, David A, Themis M, Waddington S,Knapton HJ, Miah M, Cook T, Lawrence L, Nivsarkar M,Rodeck C, Coutelle C (2003) Widespread and efficientmarker gene expression in the airway epithelia of fetal sheepafter minimally invasive tracheal application of recombinantadenovirus in utero. Gene Ther in press.Peebles DM, Wyatt JS (2002) Synergy between antenatalexposure to infection and intrapartum events in causation ofperinatal brain injury at term. BJOG 109, 737-739.Pfendner EG, Nakano A, Pulkkinen L, Christiano AM, Uitto J(2003) Prenatal diagnosis for epidermolysis bullosa: a studyof 144 consecutive pregnancies at risk. Prenat Diagn 23,447-456.Phillips JH, Hori T, Nagler A, Bhat N, Spits H, Lanier LL (1992)Ontogeny of human natural killer (NK) cells: fetal NK cellsmediate cytolytic function and express cytoplasmic CD3epsilon, delta proteins. J Exp Med 175, 1055-1066.Piedrahita JA (2000) Targeted modification of the domesticanimal genome. Theriogeniology 53, 105-116.Pitt BR, Schwarz MA, Pilewski JM, Nakayama D, Mueller GM,Robbins PD, Watkins SA, Albertine KH, Bland RD (1995)Retrovirus-mediated gene transfer in lungs of living fetalsheep. Gene Ther 2, 344-350.Plopper CG, Weir AJ, Nishio SJ, Cranz DL, St George JA (1986)Tracheal submucosal gland development in the rhesusmonkey, Macaca mulatta: ultrastructure and histochemistry.Anat Embryol 174, 167-178.Ponder KP, Melniczek JR, Xu L, Weil MA, O'Malley TM,O'Donnell PA, Knox VW, Aguirre GD, Mazrier H,Ellinwood NM, Sleeper M, Maguire AM, Volk SW, MangoRL, Zweigle J, Wolfe JH, Haskins ME (2002) Therapeuticneonatal hepatic gene therapy in mucopolysaccharidosis VIIdogs. Proc Natl Acad Sci 99, 13102-13107.206
Gene Therapy and Molecular Biology Vol 7, page 207Porada CD, Tran N, Eglitis M, Moen RC, Troutman L, FlakeAW, Zhao Y, Anderson WF, Zanjani ED (1998) In uterogene therapy: transfer and long-term expression of thebacterial neo r gene in sheep after direct injection of retroviralvectors into preimmune fetuses. Hum Gene Ther 9, 1571-1585.Preece MA, Green A (2002) Pregnancy and inherited metabolicdisorders: maternal and fetal complications. Ann ClinBiochem 39, 444-455.Quintero RA, Reich H, Puder KS, Bardicef M, Evans MI,CottonDB, Romero R (1994) Umbilical-Cord Ligation of anAcardiac Twin by Fetoscopy at 19 Weeks of Gestation. NEngl J Med 330, 469-471.Raftopoulos H, Ward M, Leboulch P, Bank A (1997) Long-termtransfer and expression of the human _-globin gene in amouse transplant model. Blood 90, 3414-3422.Raper SE, Yudkoff M, Chirmule N, Gao GP, Nunes F, HaskalZJ, Furth EE, Propert KJ, Robinson MB, Magosin S, SimoesH, Speicher L, Hughes J, Tazelaar J, Wivel NA, Wilson JM,Batshaw ML (2002) A pilot study of in vivo liver-directedgene transfer with an adenoviral vector in partial ornithinetranscarbamylase deficiency. Hum Gene Ther 13, 163-175.Recombinant DNA Advisory Committee (2000) Prenatal genetransfer; Scientific, medical, and ethical issues. Hum GeneTher 11, 1211-1229.Riley RL (1998) Neonatal immune response. In: Delves PJ, RoittIM (Eds.), Encyclopedia of Immunology. Academic Press,London, pp. 1818-1821.Rodeck C (1980) Fetoscopy guided by real-time ultrasound forpure fetal blood samples, fetal skin samples, and examinationof the fetus in utero. BJOG 87, 449-456.Rodeck CH, Deans A (1999) Red cell alloimmunisation. In:Rodeck CH, Whittle MJ (Eds.), Fetal medicine: basicscience and clinical practice. Churchill Livingstone,London, pp. 785-804.Ross J, Jurkovic D, Nicolaides KH (1997) Coelocentesis: a studyof short-term safety. Prenat Diagn 17, 913-917.Russel DW, Berger MS, Miller AD (1995) The effects of humanserum and cerebrospinal fluid on retroviral vectors andpackaging cell lines. Hum Gene Ther 6, 635-641.Sabatino DE, MacKenzie TC, Campagnoli C, Liu Y-L, High KA,Flake AW (2002) Long-term expression of factor IX after inutero administration of adeno-associated viral vectors inhemophilia B mice. Blood 100 (11), 3421Sadelain M (2002) Globin gene transfer for the treatment ofsevere hemoglobinopathies: a paradigm for stem cell-basedgene therapy. J Gene Med 4, 113-121.Santolaya-Forgas J, Vengalil S, Kushwaha A, Bieniarz A,Fortman J (1998) Assessment of the risk of fetal loss afterthe coelocentesis procedure using a baboon model. FetalDiagn Ther 13, 257-260.Sarzotti M, Robbins DS, Hoffman PM (1996) Induction ofprotective CTL responses in newborn mice by a murineretrovirus. Science 271, 1726-1728.Schachtner SK, Buck CA, Bergelson JM, Baldwin HS (1996)Temporally regulated expression patterns following in uteroadenovirus-mediated gene transfer. Gene Ther 6, 1249-1257.Schiedner G, Morral N, Parks RJ, Wu Y, Koopmans SC,Langston C, Graham FL, Beaudet AL, Kochanek S (1998)Genomic DNA transfer with a high-capacity adenovirusvector results in improved in vivo gene expression anddecreased toxicity. Nat Genet 18, 180-183.Schneider H, Adebakin S, Themis M, Cook T, Douar A-M,Pavirani A, Coutelle C (1999) Therapeutic plasmaconcentrations of human factor IX in mice after genedelivery into the amniotic cavity: a model for the prenataltreatment of haemophilia B. J Gene Med 1, 424-432.Schneider H, Coutelle C (1999) In utero gene therapy: The casefor. Nat Med 5, 256-257.Schneider H, Mühle C, Douar A-M, Waddington S, Jiang Q-J,von der Mark K, Coutelle C, Rascher W (2002) Sustaineddelivery of therapeutic concentrations of human clottingfactor IX - a comparison of adenoviral and AAV vectorsadministered in utero. J Gene Med 4, 46-53.Sekhon HS, Larson JE (1995) In utero gene transfer into thepulmonary epithelium. Nat Med 1, 1201-1203.Senoo M, Matsubara Y, Fujii K, Nagasaki Y, Hiratsuka M, KureS, Uehara S, Okamura K, Yajima A, Narisawa K (2000)Adenovirus-mediated in utero gene transfer in mice andguinea pigs: tissue distribution of recombinant adenovirusdetermined by quantitative TaqMan-polymerase chainreaction assay. Mol Genet Metab 69, 269-276.Shiotani A, Fukumura M, Maeda M, Hou X, Inoue M, KanamoriT, Komaba S, Washizawa K, Fujikawa S, Yamamoto T,Kadono C, Watabe K, Fukuda H, Saito K, Sakai Y, Nagai Y,Kanzaki J, Hawegawa M (2001) Skeletal muscleregeneration after insulin-like growth factor 1 gene transferby recombinant Sendai virus vector. Gene Ther 8, 1043-1050.Silverstein AM, Prendergast RA, Kraner KL (1964) Fetalresponse to antigenic stimulus IV. Rejection of skinhomografts by the fetal lamb. J Exp Med 119, 955-964.Silverstein AM, Uhr JW, Lukes RJ (1963) Fetal response toantigenic stimulus II. Antibody production by the fetal lamb.J Exp Med 117, 799-812.Simonds AK, Ward S, Heather S, Bush A, Muntoni F (2000)Outcome of paediatric domiciliary mask ventilation inneuromuscular and skeletal disease. Eur Respir J 16, 476-481.Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin H-F,Stafford DW, Patel S, Thompson AR, Nichols T, Read MS,Brinkhous DM, Kay MA (1999) Correction of hemophilia Bin canine and murine models using recombinant adenoassociatedviral vectors. Nat Med 5, 64-70.Soucie JM, Nuss R, Evatt B, Abdelhak A, Cowan L, Jill H,Kolakoski M, Wilber N (2000) Mortality among males withhemophilia: relations with source of medical care. TheHemophilia Surveillance System Project Investigators. Blood96, 437-442.Stecenko AA, Brigham KL (2003) Gene therapy progress andprospects: alpha-1 antitrypsin. Gene Ther 10, 95-99.Stratford-Perricaudet L, Levrero M, Chasse J-F, Perricaudet M,Briand P (1990) Evaluation of the transfer and expression inmice of an enzyme encoding gene using a human adenovirusvector. Hum Gene Ther 1, 241-256.Summar M, Tuchman M (2001) Proceedings of a consensusconference for the management of patients with urea cycledisorders. J Pediatr 138, S6-S10.Sun L, Li J, Xiao X (2000) Overcoming adeno-associated virusvector size limitation through viral DNA heterodimerization.Nat Med 6, 599-602.Sylvester KG, Yang EY, Cass DL, Crombleholme TM, ScottAdzick N (1997) Fetoscopic gene therapy for congenital lungdisease. J Pediatr Surg 7, 964-969.Tan LK, Kumar S, Jolly M, Gleeson C, Johnson P, Fisk N (2003)Test amnioinfusion to determine suitability for serialtherapeutic amnioinfusion in midtrimester premature ruptureof membranes. Fetal Diagn Ther 18, 183-189.Tarantal AF (1990) Interventional ultrasound in pregnantmacaques: embryonic/fetal applications. J Med Primatol 19,47-58.Tarantal AF, Lee CI, Ekert JE, McDonald R, Kohn DB, PlopperCG, Case SS, Bunnell BA (2001a) Lentiviral vector genetransfer into fetal rhesus monkeys (Macaca mulatta): lungtargetingapproaches. Mol Ther 4, 614-621.207
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