Gene therapy for mucopolysaccharidosis

Expert Opinion on Biological Therapy

Volumn 7, Issue 9, 2007, Pages 1333-1345

  Ponder, Katherine P ^a   Haskins, Mark E ^b  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Gene therapy; Glycosaminoglycan; Lysosomal storage disease; Mucopolysaccharidosis

Indexed keywords

ADENOVIRUS VECTOR; BETA ACTIN; BETA GLUCURONIDASE; PARVOVIRUS VECTOR; PHOSPHOGLYCEROL KINASE; PHOSPHOTRANSFERASE; PLASMID VECTOR; RETROVIRUS VECTOR; TRYPSIN INHIBITOR; UNCLASSIFIED DRUG;

DRUG EFFECT; DRUG EFFICACY; DRUG MEGADOSE; ENZYME ACTIVITY; ENZYME RELEASE; EX VIVO GENE TRANSFER; HEMATOPOIETIC STEM CELL; IN VIVO GENE TRANSFER; LEUKEMIA; LIVER TUMOR; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PROTEIN EXPRESSION; REVIEW; VIRAL GENE THERAPY;

ANIMALS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLUCURONIDASE; HUMANS; MUCOPOLYSACCHARIDOSES;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA;

EID: 34748824391     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.7.9.1333     Document Type: Review

References (103)

1. NEUFELD ER MUENZER J: The mucopolysaccharidoses. In: Metabolic and Molecular Bask of Inherited Disease. Scriver CR, Beaudet AL, Sly WS, Valle D (Eds), McGraw Hill: New York, USA (2001):3421-3452.
2. BAEHNER F, SCHMIEDESKAMP C, KRUMMENAUER F et al.: Cumulative incidence rates of the mucopolysaccharidoses in Germany. J. Inherit. Metab. Dis. (2005) 28(6):1011-1017.
3. DESNICK RJ: Enzyme replacement and enhancement therapies for lysosomal diseases. J. Inherit. Metab. Dis. (2004) 27(3):385-410.
4. HOOGERBRUGGE PM, BROUWER OF, BORDIGONI P et al.: Allogeneic bone marrow transplantation for lysosomal storage diseases. The European group for bone marrow transplantation. Lancet (1995) 345(8962): 1398-1402.
5. SIFUENTES M, DOROSHOW R, HOFT R: A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol. Genet. Metab. (2007) 90(2):171-180.
6. MUENZER J, WRAITH JE, BECK M et al.: A Phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet. Med. (2006) 8(8):465-473.
7. HARMATZ P, GIUGLIANI R, SCHWARTZ I et al.: Enzyme replacement therapy for mucopolysaccharidosis VI: a Phase III, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J. Pediatr. (2006) 148(4):533-539.
8. PETERS C, SHAPIRO EG, ANDERSON J et al.: Hurler syndrome. II. Outcome of HLA-genotypically identical sibling and HLA-haploidemical related donor bone marrow transplantation in fifty-four children. The storage disease collaborative study group. Blood (1998) 91(7):2601-2608.
9. STABA SL, ESCOLAR ML, POE M et al.: Cord-blood transplants from unrelated donors in patients with Hurlers syndrome. N. Engl. J. Med. (2004) 350(19):1960-1969.
10. VELLODI A, YOUNG E, COOPER A, LIDCHI V, WINCHESTER B, WRAITH JE: Long-term follow-up following bone marrow transplantation for Hunter disease. J. Inherit. Metab. Dis. (1999) 22(5):638-648.
11. ERSKHOVITZ E, YOUNG E, RAINER J et al.: Bone marrow transplantation for Maroteaux-Lamy syndrome (MPS VI): long-term follow-up. J. Inherit. Metab. Dis. (1999) 22(1):50-62.
12. SARDIELLO M, ANNUNZIATA I, ROMA G, BALLABIO A: Sulfatases and sulfatase modifying factors: an exclusive and promiscuous relationship. Hum. Mol. Genet. (2005) 14(21):3203-3217.
13. FRALDI A, BIFFI A, LOMBARDI A et al.: SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. Biochem. J. (2007) 403(2):305-312.
14. CARDONE M, POLITO VA, PEPE S et al.: Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery. Hum. Mol. Genet. (2006) 15(7):1225-1236.
15. YOUNG LS, SEARLE PF, ONION D, MAUTNER V: Viral gene therapy strategies: from basic science to clinical application. J. Pathol. (2006) 208(2):299-318.
16. HODGES BL, CHENG SH: Cell and gene-based therapies for the lysosomal storage diseases. Curr. Gene Ther. (2006) 6(2):227-241.
17. ELLINWOOD NM, VITE CH, HASKINS ME: Gene therapy for lysosomal storage diseases: the lessons and promise of animal models. J. Gene Med. (2004) 6(5):481-506.
18. CHEN ZY, HE CY, EHRHARDT A, KAY MA: Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo Mol. Ther. (2003) 8(3):495-500.
19. ARONOVICH EL, BELL JB, BELUR LR et al.: Prolonged expression of a lysosomal enzyme in mouse liver after sleeping beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses. J. Gene Med. (2007) 9(5):403-415.
20. SANDS MS, DAVIDSON BL: Gene therapy for lysosomal storage diseases. Mol. Ther. (2006) 13(5):839-849.
21. XU L, MANGO RL, SANDS MS, HASKINS ME, ELLINWOOD NM, PONDER KP: Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Mol. Ther. (2002) 6(6):745-758.
22. LIU Y, XU L, HENNIG AK et al.: Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice. Mol. Ther. (2005) 11(1):35-47.
23. CHUNG S, MA X, LIU Y, LEE D, TITTIGER M, PONDER KP: Effect of neonatal administration of a retroviral vector expressing α-1-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice. Mol. Genet. Metabol. (2007) 90(2):181-192.
24. DALY TM, OHLEMILLER KK, ROBERTS MS, VOGLER CA, SANDS MS: Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Ther. (2001) 8(17):1291-1298.
25. DALY TM, VOGLER C, LEVY B, HASKINS ME, SANDS MS: Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc. Natl. Acad. Sci. USA (1999) 96(5):2296- 2300.
26. DONSANTE A, LEVY B, VOGLER C, SANDS MS: Clinical response to persistent, low-level β-glucuronidase expression in the murine model of mucopolysaccharidosis type VII. J. Inherit. Metab. Dis. (2007) 30(2):227-238.
27. HARTUNG SD, FRANDSEN, JL, PAN D et al.: Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-L-iduronidase gene. Mol. Ther. (2004) 9(6):866-875.
28. MA X, LIU Y, TITTIGER M et al.: Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation. Mol. Ther. (2007) 15:889-902.
29. PONDER KP, MELNICZEK JR, XU L et al.: Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proc. Natl. Acad. Sci. USA (2002) 99(20):13102-13107.
30. SLEEPER MM, FORNASARI B, ELLINWOOD NM et al.: Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII. Circulation (2004) 110(7):815-820.
31. MANGO RL, XU L, SANDS MS et al.: Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs. Mol. Genet. Metab. (2004) 82(1):4-19.
32. WANG B, O MALLEY TM, XU L et al.: Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs. Mol. Genet. Metab. (2006) 87(1):8-21.
33. TRAAS AM, WANG P, MA X et al.: Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy. Mol. Ther. 33:1423-1431.
34. SFERRA TJ, BACKSTROM K, WANG C, RENNARD R, MILLER M, HU Y: Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse. Mol. Ther. (2004) 10(3):478-491.
35. DALY TM, OKUYAMA T, VOGLER C, HASKINS ME, MUZYCZKA N, SANDS MS: β-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. Hum. Gene Ther. (1999) 10(1):85-94.
36. FRISO A, TOMANIN R, ALBA S et al.: Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts. J. Gene Med. (2005) 7(11):1482-1491.
37. ROSS CJ, BASTEDO L, MAIER SA, SANDS MS, CHANG PL: Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells. Hum. Gene Ther. (2000) 11(15):2117-2127.
38. MOULLIER P, BOHL D, HEARD JM, DANOS O: Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts. Nat. Genet. (1993) 4(2):154-159.
39. WOLFE JH, SANDS MS, HAREL N et al.: Gene transfer of low levels of β-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII. Mol. Ther. (2000) 2(6):552-561.
40. SHULL RM, LU X, MCENTEE MF, BRIGHT RM, PEPPER KA, KOHN DB: Myoblast gene therapy in canine mucopolysaccharidosis. I: abrogation by an immune response to α-L-iduronidase. Hum. Gene Ther. (1996) 7(13):1595-1603.
41. WOLFE JH, SANDS MS, BARKER JE et al.: Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature (1992) 360(6406):749-753.
42. + hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. Mol. Ther. (2004) 9(6):856-865.
43. ZHENG Y, ROZENGURT N, RYAZANTSEV S, KOHN DB, SATAKE N, NEUFELD EF: Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Mol. Genet. Metab. (2003) 79(4):233-244.
44. ZHENG Y, RYAZANTSEV S, OHMI K et al.: Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol. Genet. Metab. (2004) 82(4):286-295.
45. LUTZKO C, KRUTH S, ABRAMS-OGG AC et al.: Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine α-L-iduronidase deficiency. Blood (1999) 93(6):1895-1905.
46. LUTZKO C, OMORI F, ABRAMS-OGG AC et al.: Gene therapy for canine α-L-iduronidase deficiency: in utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease. Hum. Gene Ther. (1999) 10(9):1521-1532.
47. MEERTENS L, ZHAO Y, ROSIC-KABLAR S et al.: In utero injection of α-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. Hum. Gene Ther. (2002) 13(15):1809-1820.
48. MILLER DG, WANG PR, PETEK LM, HIRATA RK, SANDS MS, RUSSELL DW: Gene targeting in vivo by adeno-associated virus vectors. Nat. Biotechnol. (2006) 24(8):1022-1026.
49. VOGLER C, LEVY B, GRUBB JH et al.: Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII. Proc. Natl. Acad. Sci. USA (2005) 102(41):14777-14782.
50. KOBAYASHI H, CARBONARO D, PEPPER K et al.: Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol. Ther. (2005) 11:776-789.
51. HENNIG AK, LEVY B, OGILVIE JM et al.: Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice. J. Neurosci. (2003) 23(8):3302-3307.
52. CHEN F, VITRY S, HOCQUEMILLER M, DESMARIS N, AUSSEIL J, HEARD JM: α-L-iduronidase transport in neuntes. Mol. Genet. Metab. (2006) 87(4):349-358.
53. PASSINI MA, LEE EB, HEUER GG, WOLFE JH: Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. J. Neurosci. (2002) 22(15):6437-6446.
54. XIA H, MAO Q, DAVIDSON BL: The HIV Tat protein transduction domain improves the biodistribution of β-glucuronidase expressed from recombinant viral vectors. Nat. Biotechnol. (2001) 19(7):640-644.
55. DESMARIS N, VEROT L, PUECH JP, CAILLAUD C, VANIER MT, HEARD JM: Prevention of neuropathology in the mouse model of Hurler syndrome. Ann. Neurol (2004) 56(1):68-76.
56. CRESSANT A, DESMARIS N, VEROT L et al.: Improved behavior and neuropathology in the mouse model of Sanfilippo type IHB disease after adeno-associated virus-mediated gene transfer in the striatum. J. Neurosci. (2004) 24(45): 10229-10239.
57. BOSCH A, PERRET E, DESMARIS N, HEARD JM: Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors. Mol. Ther. (2000) 1(1):63-70.
58. LIU G, CHEN YH, HE X et al.: Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS. Mol. Ther. (2007) 15(2):242-247.
59. STEIN CS, GHODSI A, DERKSEN T, DAVIDSON BL: Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice. J. Virol. (1999) 73(4):3424-3429.
60. BROOKS AI, STEIN CS, HUGHES SM et al.: Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors. Proc. Natl. Acad. Sci. USA (2002) 99(9):6216-6221.
61. VITE CH, PASSINI MA, HASKINS ME, WOLFE JH: Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. (2003) 10(22):1874-1881.
62. PASSINI MA, WATSON DJ, VITE CH, LANDSBURG DJ, FEIGENBAUM AL, WOLFE JH: Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of β-glucuronidase-deficient mice. J. Virol. (2003) 77(12):7034-7040.
63. LIU G, MARTINS I, WEMMIE JA, CHIORINI JA, DAVIDSON BL: Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. J. Neurosci. (2005) 25(41):9321-9327.
64. 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. (2006) 14(1):14-24.
65. GHODSI A, STEIN C, DERKSEN T, MARTINS I, ANDERSON RD, DAVIDSON BL: Systemic hyperosmolality improves β-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer. Exp. Neurol. (1999) 160(1):109-116.
66. OHASHI T, WATABE K, UEHARA K, SLY WS, VOGLER C, ETO Y: Adenovirus-mediated gene transfer and expression of human β-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc. Natl. Acad. Sci. USA (1997) 94(4):1287-1292.
67. SAKURAI K, IIZUKA S, SHEN JS et al.: Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice. Gene Ther. (2004) 11(19):1475-1481.
68. FU H, SAMULSKI RJ, MCCOWN TJ, PICORNELL YJ, FLETCHER D, MUENZER J: Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery. Mol. Ther. (2002) 5(1):42-49.
69. BERGES BK, YELLAYI S, KAROLEWSKI BA, MISELIS RR, WOLFE JH, FRASER NW: Widespread correction of lysosomal storage in the mucopolysaccharidosis type VII mouse brain with a herpes simplex virus type 1 vector expressing β-glucuronidase. Mol. Ther. (2006) 13(5):859-869.
70. BOSCH A, PERRET E, DESMARIS N, TRONO D, HEARD JM: Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum. Gene Ther. (2000) 11(8):1139-1150.
71. FRISELLAWA, O'CONNOR LH, VOGLER C et al.: Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Mol Ther. (2001) 3(3):351-358.
72. WATSON G, BASTACKY J, BELICHENKO P et al.: Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice. Gene Ther. (2006) 13(11):917-925.
73. ELLIGER SS, ELLIGER CA, AGUILAR CP, RAJU NR, WATSON GL: Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector. Gene Ther. (1999) 6(6):1175-1178.
74. CIRON C, DESMARIS N, COLE MA et al: Gene therapy of the brain in the dog model of Hurler s syndrome. Ann. Neurol. (2006) 60(2):204-213.
75. HENNIG AK, OGILVIE JM, OHLEMILLER KK, TIMMERS AM, HAUSWIRTH WW, SANDS MS: AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice. Mol. Ther. (2004) 10(1):106-116.
76. KAMATA Y, OKUYAMA T, KOSUGA M et al.: Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII. Mol. Ther. (2001) 4(4):307-312.
77. LIT, DAVIDSON BL: Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer. Proc. Natl. Acad. Sci. USA (1995) 92(17):7700-7704.
78. HO TT, MAGUIRE AM, AGUIRRE GD et al.: Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI. J. Gene Med. (2002) 4(6):613-621.
79. MATTE U, YOGALINGAM G, BROOKS D et al.: Identification and characterization of 13 new mutations in mucopolysaccharidosis type I patients. Mol. Genet. Metab. (2003) 78(1):37-43.
80. PONDER KP, WANG B, WANG P P et al.: Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig. Mol. Ther. (2006) 14(1):5-13.
81. DI DOMENICO C, VILLANI GR, DI NAPOLI D et al.: Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum. Gene Ther. (2005) 16(1):81-90.
82. DI DOMENICO C, DI NAPOLI D, GONZALEZ Y et al.: Limited transgene immune response and long-term expression of human α-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy. Hum. Gene Ther. (2006) 17(11):1112-1121.
83. BROWN BD, VENNERI MA, ZINGALE A, SERGI SERGI L, NALDINI L: Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat. Med. (2006) 12(5):585-591.
84. GAO C, SANDS MS, HASKINS ME, PONDER KP: Delivery of a retroviral vector expressing human β-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice. Mol. Ther. (2000) 2(3):233-244.
85. KOSUGA M, TAKAHASHI S, SASAKI K et al.: Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression. Mol. Ther. (2000) 1(5):406-413.
86. BAILEY SI, HOWE SJ, SCHAMBACH A, KINNON C, BAUM C, THRASHER AJ: Novel γ-retroviral vectors for gene therapy of SCID-X1. Mol. Ther. (2006) 13:5256.
87. NIENHUIS AW, DUNBAR CE, SORRENTINO BP: Genotoxicity of retroviral integration in hematopoietic cells. Mol. Ther. (2006) 13(6):1031-1049.
88. THEMIS M, WADDINGTON SN, SCHMIDT M et al.: Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol. Ther. (2005) 12(4):763-771.
89. MODLICH U, BOHNE J, SCHMIDT M et al.: Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood (2006) 108(8):2545-2553.
90. MONTINI E, CESANA D, SCHMIDT M et al.: Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat. Biotechnol. (2006) 24(6):687-696.
91. CALOS MP: The phiC31 integrase system for gene therapy. Curr. Gene Ther. (2006) 6(6):633-645.
92. DONSANTE A, VOGLER C, MUZYCZKA N et al.: Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther. (2001) 8(17):1343-1346.
93. DONSANTE A, MILLER DG, LI Y et al.: AAV vector integration sites in mouse hepatocellular carcinoma. Science 317:477.
94. MALIK P, ARUMUGAM PI, YEE JK, PUTHENVEETIL G: Successful correction of the human Cooley's anemia {β}-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator. Ann. NY Acad. Sci. (2005) 1054:238-249.
95. BLUMENTHAL M, SKELTON D, PEPPER KA, JAHN T, METHANGKOOL E, KOHN DB: Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Mol. Ther. (2007) 15(1):183-192.
96. POWELL JS, RAGNI MV, WHITE GC et al.: Phase I trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood (2003) 102(6):2038-2045.
97. MANNO CS, PIERCE GF, ARRUDA VR et al.: Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat. Med. (2006) 12(3):342-347.
98. MEIKLE PJ, GRASBY DJ, DEAN CJ et al.: Newborn screening for lysosomal storage disorders. Mol. Genet. Metab. (2006) 88(4):307-314.
99. WANG D, EADALA B, SADILEK M et al.: Tandem mass spectrometric analysis of dried blood spots for screening of mucopolysaccharidosis I in newborns. Clin. Chem. (2005) 51(5):898-900.
100. ANSON DS, MCINTYRE C, THOMAS B et al.: Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA. Genet. Vaccines Ther. (2007) 5:1.
101. ELLIGER SS, ELLIGER CA, LANG C, WATSON GL: Enhanced secretion and uptake of β-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice. Mol. Ther. (2002) 5(5):617-626.
102. KAMATA Y, TANABE A, KANAJI A et al.: Long-term 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. (2003) 10(5):406-414.
103. KANAJI A, KOSUGA M, LI XK et al.: Improvement of skeletal lesions in mice with mucopolysaccharidosis type VII by neonatal adenoviral gene transfer. Mol. Ther. (2003) 8(5):718-725.