Recent advances in fetal gene therapy

Therapeutic Delivery

Volumn 2, Issue 4, 2011, Pages 461-469

  Buckley, Suzanne M K ^a   Rahim, Ahad A ^a   Chan, Jerry K Y ^b   David, Anna L ^a   Peebles, Donald M ^a   Coutelle, Charles ^c   Waddington, Simon N ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b NATIONAL UNIVERSITY OF SINGAPORE   (Singapore)

^c NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALPHA GLOBIN; ALPHA GLUCOSIDASE; BETA GLUCURONIDASE; BILIRUBIN; BLOOD CLOTTING FACTOR 7; BLOOD CLOTTING FACTOR 9; DYSTROPHIN; FIBROBLAST GROWTH FACTOR 10; GENE PRODUCT; GLUCURONOSYLTRANSFERASE; LENTIVIRUS VECTOR; LEVO IDURONIDASE; PARVOVIRUS VECTOR; PROTEIN C; RETINA PIGMENT EPITHELIUM 65; RETROVIRUS VECTOR; TRANSFORMING GROWTH FACTOR BETA1; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR CLEAVING PROTEINASE;

ADENO ASSOCIATED VIRUS; ALPHA THALASSEMIA; ARTICLE; BILIRUBIN BLOOD LEVEL; BLOOD CLOTTING FACTOR 7 DEFICIENCY; CRIGLER NAJJAR SYNDROME; CYSTIC FIBROSIS; DEVELOPMENTAL BIOLOGY; DISEASE MODEL; DUCHENNE MUSCULAR DYSTROPHY; ELECTROPORATION; EMBRYO; FETAL GENE THERAPY; FETUS; GAUCHER DISEASE; GENE THERAPY; GENE TRANSFER; GLYCOGEN STORAGE DISEASE TYPE 2; HEMOPHILIA B; HUMAN; HURLER SYNDROME; LEBER CONGENITAL AMAUROSIS; LIVER CELL CARCINOMA; LUNG CYST; LUNG FIBROSIS; LUNG HYPOPLASIA; MEDICAL RESEARCH; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PRIORITY JOURNAL; PROTEIN C DEFICIENCY; THALASSEMIA; THROMBOTIC THROMBOCYTOPENIC PURPURA; TRANSGENICS; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM;

EID: 79954548022     PISSN: 20415990     EISSN: 20416008     Source Type: Journal    
DOI: 10.4155/tde.11.17     Document Type: Article

References (70)

1. Watanabe M, Flake AW. Fetal surgery: progress and perspectives. Adv. Pediatr. 57(1), 353-372 (2010).
2. Pollock-Barziv SM, Mccrindle BW, West LJ, Dipchand AI. Waiting before birth: outcomes after fetal listing for heart transplantation. Am. J. Transplant. 8(2), 412-418 (2008).
3. Seppen J, Van Der Rijt R, Looije N, Van Til NP, Lamers WH, Oude Elferink RP. Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer. Mol. Ther. 8(4), 593-599 (2003). (Pubitemid 37321688)
4. Waddington SN, Nivsarkar M, Mistry A et al. Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy. Blood 104(9), 2714-2721 (2004). (Pubitemid 39434952)
5. Niiya M, Endo M, Shang D et al. Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes. Mol. Ther. 17(1), 34-41 (2009).
6. Banno F, Kokame K, Okuda T et al. Complete deficiency in ADAMTS13 is prothrombotic, but it alone is not sufficient to cause thrombotic thrombocytopenic purpura. Blood 107(8), 3161-3166 (2006).
7. Themis M, Waddington SN, Schmitt M et al. Oncogenesis following delivery of a non-primate lentiviral gene therapy vector to fetal and neonatal mice. Mol. Ther. 12(4), 763-771 (2005). (Pubitemid 41350158)
8. Porada CD, Park PJ, Tellez J et al. Male germ-line cells are at risk following direct-injection retroviral-mediated gene transfer in utero. Mol. Ther. 12(4), 754-762 (2005). (Pubitemid 41350157)
9. Park PJ, Colletti E, Ozturk F et al. Factors determining the risk of inadvertent retroviral transduction of male germ cells after in utero gene transfer in sheep. Hum. Gene Ther. 20(3), 201-215 (2009).
10. Lee CC, Jimenez DF, Kohn DB, Tarantal AF. Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta).Hum. Gene Ther. 16(4), 417-425 (2005). (Pubitemid 40647139)
11. Dejneka NS, Surace EM, Aleman TS et al. In utero gene therapy rescues vision in a murine model of congenital blindness. Mol. Ther. 9(2), 182-188 (2004). (Pubitemid 38256544)
12. Karolewski BA, Wolfe JH. Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol. Ther. 14(1), 14-24 (2006). (Pubitemid 43899484)
13. Rucker M, Fraites TJ Jr, Porvasnik SL et al. Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development 131(12), 3007-3019 (2004). (Pubitemid 38997287)
14. Sabatino DE, Mackenzie TC, Peranteau W et al. Persistent expression of hF.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B Mice. Mol Ther 12, 12 (2007).
15. Koppanati BM, Li J, Reay DP et al. Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene. Gene Ther. 17(11), 1355-1362 (2010).
16. Koppanati BM, Li J, Xiao X, Clemens PR. Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders. Gene Ther. 16(9), 1130-1137 (2009).
17. Donsante A, Miller DG, Li Y et al. AAV vector integration sites in mouse hepatocellular carcinoma. Science 317(5837), 477 (2007). (Pubitemid 47196106)
18. Nathwani AC, Rosales C, Mcintosh J et al. Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol. Ther. DOI:10.1038/ mt.2010.274 (2011) (Epub ahead of print).
19. Larson JE, Morrow SL, Happel L, Sharp JF, Cohen JC. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 349(9052), 619-620 (1997). (Pubitemid 27101894)
20. Colledge WH. Gene therapy for cystic fibrosis. Lancet 349(9060), 1249; author reply 1250 (1997).
21. Buckley SMK, Waddington SN, Jezzard S et al. Intra-amniotic delivery of CFTR expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice. Mol. Ther. 16(5), 819-824 (2008). (Pubitemid 351587089)
22. Davies LA, Varathalingam A, Painter H et al. Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice. Mol. Ther. 16(5), 812-818 (2008). (Pubitemid 351587088)
23. Meertens L, Zhao Y, Rosic-Kablar S et al. In utero injection of a-l-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. Hum. Gene Ther. 13(15), 1809-1820 (2002). (Pubitemid 35292678)
24. Han XD, Lin C, Chang J, Sadelain M, Kan YW. Fetal gene therapy of a-thalassemia in a mouse model. Proc. Natl Acad. Sci. USA 104(21), 9007-9011 (2007). (Pubitemid 47175427)
25. Kantoff PW, Flake AW, Eglitis MA et al. In utero gene transfer and expression: a sheep transplantation model. Blood 73(4), 1066-1073 (1989). (Pubitemid 19078390)
26. Flake AW, Roncarolo MG, Puck JM et al. Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. N. Engl. J. Med. 335(24), 1806-1810 (1996). (Pubitemid 26412399)
27. Colletti E, Lindstedt S, Park PJ, Almeida- Porada G, Porada CD. Early fetal gene delivery utilizes both central and peripheral mechanisms of tolerance induction. Exp. Hematol. 36(7), 816-822 (2008).
28. David A, Cook T, Waddington S et al. Ultrasound guided percutaneous delivery of adenoviral vectors encoding the b-galactosidase and human factor IX genes to early gestation fetal sheep in utero. Hum. Gene Ther. 14(4), 353-364 (2003). (Pubitemid 36308354)
29. David AL, Torondel B, Zachary IC et al. Local delivery of VEGF adenovirus to the uterine artery increases vasorelaxation and blood flow in the pregnant sheep. Gene Ther. 15(19), 1344-1350 (2008).
30. David AL, Mcintosh J, Peebles DM et al. rAAV mediates in utero gene transfer gives therapeutic transgene expression in the sheep. Hum. Gene Ther. DOI:10.1089/ hum.2010.007 (2011) (Epub ahead of print).
31. Tarantal AF, Lee C. Long-term luciferase expression monitored by bioluminescence imaging after AAV-mediated fetal gene delivery in Rhesus monkeys (Macaca mulatta).Hum. Gene Ther. 21, 1-6 (2010).
32. Tarantal AF, Chen H, Shi TT et al. Overexpression of transforming growth factor-b1 in fetal monkey lung results in prenatal pulmonary fibrosis. Eur. Respir. J. 36(4), 907-914 (2010).
33. Tarantal AF, Lee CCI, Jimenez DF, Cherry SR. Fetal gene transfer using lentiviral vectors: in vivo detection of gene expression by microPET and optical imaging in fetal and infant monkeys. Hum. Gene Ther. 17, 1254-1261 (2006). (Pubitemid 44975141)
34. Mattar CN, Dighe NM, Waddington SN et al. Long-term transgene expression in new-born cynomolgus macaques following intra-uterine gene transfer of self-complementary AAV. Mol. Ther. (2011) (In press).
35. Mattar CN, Johana N, Fisk A et al. Late gestation intrauterine gene transfer of AAV9 in non-human primates preferentially transduces the central nervous system, liver, skeletal and cardiac musculature. Mol. Ther. 18(Suppl. 1), S272 (2010).
36. Stuhlmann H, Cone R, Mulligan RC, Jaenisch R. Introduction of a selectable gene into different animal tissue by a retrovirus recombinant vector. Proc. Natl Acad. Sci. USA 81(22), 7151-7155 (1984). (Pubitemid 15216714)
37. Stitelman DH, Endo M, Bora A et al. Robust in vivo transduction of nervous system and neural stem cells by early gestational intra amniotic gene transfer using lentiviral vector. Mol. Ther. 18, 1615-1623 (2010).
38. Endo M, Henriques-Coelho T, Zoltick PW et al. The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors. Gene. Ther. 17, 61-71 (2010).
39. Henriques-Coelho T, Gonzaga S, Endo M et al. Targeted gene transfer to fetal rat lung interstitium by ultrasound-guided intrapulmonary injection. Mol. Ther. 15(2), 340-347 (2007). (Pubitemid 46111884)
40. Endo M, Zoltick PW, Peranteau WH et al. Efficient in vivo targeting of epidermal stem cells by early gestational intraamniotic injection of lentiviral vector driven by the keratin 5 promoter. Mol. Ther. 16(1), 131-137 (2007).
41. Endo M, Zoltick PW, Chung DC et al. Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector. Mol. Ther. 15(3), 579-587 (2007).
42. Beronja S, Livshits G, Williams S, Fuchs E. Rapid functional dissection of genetic networks via tissue-specific transduction and RNAi in mouse embryos. Nat. Med. 16(7), 821-827 (2010).
43. Gonzaga S, Henriques-Coelho T, Davey M et al. Cystic adenomatoid malformations are induced by localized FGF10 overexpression in fetal rat lung. Am. J. Respir. Cell. Mol. Biol. 39(3), 346-355 (2008).
44. Gubbels SP, Woessner DW, Mitchell JC, Ricci AJ, Brigande JV. Functional auditory hair cells produced in the mammalian cochlea by in utero gene transfer. Nature 455(7212), 537-541 (2008).
45. Mishra S, Wang X, Smiley N et al. Genetic modification of airway progenitors following lentiviral gene delivery to the amniotic fluid of murine fetuses. Am. J. Respir. Cell. Mol. Biol. DOI:10.1165/rcmb.2009-0235OC (2010) (Epub ahead of print).
46. Buckley SMK, Howe S, Sheard V et al. Lentiviral transduction of the developing murine lung provides efficient pseudotype and developmental stage dependent and cell-specific transgene expression. Gene Ther. 15(16), 1167-1175 (2008).
47. Carlon M, Toelen J, Van Der Perren A et al. Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. Mol. Ther. 18, 2130-2138 (2010).
48. Santore MT, Roybal JL, Flake AW. Prenatal stem cell transplantation and gene therapy. Clin. Perinatol. 36(2), 451-471, XI (2009).
49. Waddington SN, Kramer MG, Hernandez- Alcoceba R et al. In utero gene therapy: current challenges and perspectives. Mol. Ther. 11(5), 661-676 (2005). (Pubitemid 40575612)
50. David AL, Peebles D. Gene therapy for the fetus: is there a future? Best Pract. Res. Clin. Obstet. Gynaecol. 22(1), 203-218 (2007).
51. Cavazzana-Calvo M, Hacein-Bey S, De Saint Basile G et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288(5466), 669-672 (2000). (Pubitemid 30241569)
52. Cartier N, Hacein-Bey-Abina S, Bartholomae CC et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326(5954), 818-823 (2009).
53. Guillot PV, Abass O, Bassett JH et al. Intrauterine transplantation of human fetal mesenchymal stem cells from first trimester blood repairs bone and reduces fractures in osteogenesis imperfecta mice. Blood 111(3), 1717-1725 (2008). (Pubitemid 351213464)
54. Panaroni C, Gioia R, Lupi A et al. In utero transplantation of adult bone marrow decreases perinatal lethality and rescues the bone phenotype in the knockin murine model for classical, dominant osteogenesis imperfecta. Blood 114(2), 459-468 (2009).
55. Guillot PV, Cook HT, Pusey CD et al. Transplantation of human fetal mesenchymal stem cells improves glomerulopathy in a collagen type I a2-deficient mouse. J. Pathol. 214(5), 627-636 (2008). (Pubitemid 351482278)
56. Merianos DJ, Tiblad E, Santore MT et al. Maternal alloantibodies induce a postnatal immune response that limits engraftment following in utero hematopoietic cell transplantation in mice. J. Clin. Invest. 119(9), 2590-2600 (2009).
57. Nijagal A, Wegorzewska M, Jarvis E, Le T, Tang Q, Mackenzie TC. Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice. J. Clin. Invest. 121(2), 582-592 (2011).
58. Nathwani A, Tuddenham E, Rosales C et al. Early clinical trial results following administration of a low dose of a novel self complementary adeno-associated viral vector encoding human factor IX in two subjects with severe hemophilia B. Blood 116(21), A248 (2010).
59. Pien GC, Basner-Tschakarjan E, Hui DJ et al. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J. Clin. Invest. 119(6), 1688-1695 (2009).
60. Foust KD, Wang X, Mcgovern VL et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat. Biotechnol. 28, 271-274 (2010).
61. Liu H, Shafey D, Moores JN, Kothary R. Neurodevelopmental consequences of SMN depletion in a mouse model of spinal muscular atrophy. J. Neurosci. Res. 88(1), 111-122 (2010).
62. Orvisky E, Sidransky E, Mckinney CE et al. Glucosylsphingosine accumulation in mice and patients with type 2 Gaucher disease begins early in gestation. Pediatr. Res. 48(2), 233-237 (2000). (Pubitemid 30497342)
63. Goldenberg NA, Manco-Johnson MJ. Protein C deficiency. Haemophilia 14(6), 1214-1221 (2008).
64. Landau D, Rosenberg N, Zivelin A, Staretz-Chacham O, Kapelushnik J. Familial factor VII deficiency with foetal and neonatal fatal cerebral haemorrhage associated with homozygosis to Gly180Arg mutation. Haemophilia 15(3), 774-778 (2009).
65. Rosen ED, Chan JC, Idusogie E et al. Mice lacking factor VII develop normally but suffer fatal perinatal bleeding. Nature 390(6657), 290-294 (1997). (Pubitemid 27513311)
66. Binny CJ, Mcintosh JH, Tuddenham E et al. Assessing the potential of perinatal gene transfer using congenital factor VII deficiency as a model system. Blood 116(21), A247 (2010).
67. Aiuti A, Cattaneo F, Galimberti S et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 360(5), 447-458 (2009).
68. Bainbridge JW, Smith AJ, Barker SS et al. Effect of gene therapy on visual function in leber's congenital amaurosis. N. Engl. J. Med. 358(21), 2231-2239 (2008). (Pubitemid 351724452)
69. Cideciyan AV, Hauswirth WW, Aleman TS et al. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum. Gene Ther. 20(9), 999-1004 (2009).
70. Maguire AM, High KA, Auricchio A et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a Phase 1 dose-escalation trial. Lancet 374(9701), 1597-1605 (2009).