Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy

Gene Therapy

Volumn 11, Issue 14, 2004, Pages 1117-1125

  Gregory, L G ^a   Waddington, S N ^a   Holder, M V ^a   Mitrophanous, K A ^b   Buckley, S M K ^a   Mosley, K L ^a   Bigger, B W ^a   Ellard, F M ^b   Walmsley, L E ^b   Lawrence, L ^a   Al Allaf, F ^a   Kingsman, S ^b   Coutelle, C ^a   Themis, M ^a  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b OXFORD BIOMEDICA UK LTD   (United Kingdom)

Author keywords

Duchenne muscular dystrophy; EIAV lentivirus

Indexed keywords

BETA GALACTOSIDASE; CD4 ANTIGEN; CD8 ANTIGEN; LENTIVIRUS VECTOR; VIRUS ANTIBODY; VIRUS ENVELOPE PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DIAPHRAGM MUSCLE; DRUG MECHANISM; DUCHENNE MUSCULAR DYSTROPHY; EQUINE INFECTIOUS ANEMIA VIRUS; FEMALE; FETUS; GENE EXPRESSION; GENE TRANSFER; GESTATION PERIOD; HEART MUSCLE CELL; HUMAN; HUMAN CELL; IMMUNE RESPONSE; INJECTION; INTERCOSTAL MUSCLE; LONG TERM CARE; LYMPHOCYTE COUNT; MACROPHAGE; MOUSE; MUSCULAR DYSTROPHY; NEUTROPHIL; NONHUMAN; PRIORITY JOURNAL; SKELETAL MUSCLE; VESICULAR STOMATITIS VIRUS; VIRAL GENE DELIVERY SYSTEM; VIRUS TYPING; ANIMAL; GENE THERAPY; GENETIC ENGINEERING; GENETICS; IMMUNOLOGY; METABOLISM; METHODOLOGY; PREGNANCY; PRENATAL DEVELOPMENT; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

EQUIDAE; EQUINE INFECTIOUS ANEMIA VIRUS; LENTIVIRUS;

ANIMALS; BETA-GALACTOSIDASE; FEMALE; FETUS; GENE EXPRESSION; GENE THERAPY; GENETIC ENGINEERING; INFECTIOUS ANEMIA VIRUS, EQUINE; INJECTIONS; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; PREGNANCY;

EID: 4243132862     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302268     Document Type: Article

References (25)

1. Emery AEH. Duchenne Muscular Dystrophy. Oxford University Press: Oxford, 1993.
2. Simonds AK et al. Outcome of paediatric domiciliary mask ventilation in neuromuscular and skeletal disease. Eur Respir J 2000; 16: 476-481.
3. Turkel SB, Howell R, Iseri AL, Chui L. Ultrastructure of muscle in fetal Duchenne's dystrophy. Arch Pathol Lab Med 1981; 105: 414-418.
4. Yoshioka M, Yorifuji T, Mituyoshi I. Skewed X inactivation in manifesting carriers of Duchenne muscular dystrophy. Clin Genet 1998; 53: 102-107.
5. Wells DJ, Ferrer A, Wells KE. Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy Expert Rev Mol Med 2002; 4: 1-23.
6. Chamberlain JS. Gene therapy of muscular dystrophy. Hum Mol Genet 2002; 11: 2355-2362.
7. Wells DJ, Wells KE. Gene transfer studies in animals: what do they really tell us about the prospects for gene therapy in DMD? Neuromuscul Disord 2002; 12 (Suppl 1): SH-S22.
8. Lipshutz GS et al. In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression. Mol Ther 2001; 3: 284-292.
9. Waddington SN et al. In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. Blood 2003; 101: 1359-1366.
10. Kafri T et al. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997; 17: 314-317.
11. Naldini L, Verma IM. Lentiviral vectors. Adv Virus Res 2000; 55: 599-609.
12. MacKenzie TC et al. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther 2002; 6: 349-358.
13. Kobinger GP et al. Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells. Hum Gene Ther 2003; 14: 1441-1449.
14. Waddington SN, et al. Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice. Gene Therapy 2003; 10: 1234-1240.
15. Yamada K, McCarty DM, Madden VJ, Walsh CE. Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer. Biotechniques 2003; 34: 1074-1080.
16. O'Rourke JP et al. Analysis of gene transfer and expression in skeletal muscle using enhanced EIAV lentivirus vectors. Mol Ther 2003; 7: 632-639.
17. Seppen J, Barry SC, Harder B, Osborne WRA. Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin. Blood 2001; 98: 594-596.
18. Hu J, Dunbar CE. Update on haematopoietic stem cell gene transfer using non-human primate models. Curr Opin Mol Ther 2002; 4: 482-490.
19. Demaison C et al. High-level transduction and gene expression in haematopoietic repopulating cells using a human immunodeficiency [correction of immunodeficiency] virus type1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum Gene Ther 2002; 13: 803-813.
20. Mitrophanous K et al. Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Therapy 1999; 6: 1808-1818.
21. Olsen JC. Gene transfer vectors derived from equine infectious anemia virus. Gene Therapy 1998; 5: 1481-1487.
22. Stetor SR et al. Characterization of (+) strand initiation and termination sequences located at the center of the equine infectious anemia virus genome. Biochemistry 1999; 38: 3656-3667.
23. Donello JE, Loeb JE, Hope TJ. Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J Virol 1998; 72:5085-5092.
24. Martin-Rendon E et al. New methods to titrate EIAV-based lentiviral vectors. Mol Ther 2002; 5: 566-570.
25. Rohll JB et al. Design, production, safety, evaluation, and clinical applications of nonprimate lentiviral vectors. Methods Enzymol 2002;346:466-500.