In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates

BMC Biotechnology

Volumn 3, Issue , 2003, Pages

  Garrett, Deiadra J ^a,b   Larson, Janet E ^a   Dunn, Daisy ^a   Marrero, Luis ^b   Cohen, J Craig ^b  

^a OCHSNER CLINIC FOUNDATION   (United States)

^b LOUISIANA STATE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; GREEN FLUORESCENT PROTEIN; PLASMID DNA; DNA; PHOTOPROTEIN; RECOMBINANT DNA;

ADENO ASSOCIATED VIRUS; AMNION FLUID; ARTICLE; CONTROLLED STUDY; FETUS; GENE EXPRESSION; GENE TRANSFER; MOUSE; MULTIPOTENT STEM CELL; NONHUMAN; PRIMATE; PROTEIN EXPRESSION; RAT; REPORTER GENE; RODENT; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS GENE; VIRUS RECOMBINANT; ANIMAL; C57BL MOUSE; GENE VECTOR; GENETICS; INJECTION; INTESTINE; LUNG; METABOLISM; PRENATAL DEVELOPMENT; RHESUS MONKEY; SPRAGUE DAWLEY RAT; TIME;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; PRIMATES; RODENTIA;

AMNIOTIC FLUID; ANIMALS; DEPENDOVIRUS; DNA; DNA, RECOMBINANT; FETUS; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; INJECTIONS; INTESTINES; LUMINESCENT PROTEINS; LUNG; MACACA MULATTA; MICE; MICE, INBRED C57BL; RATS; RATS, SPRAGUE-DAWLEY; TIME FACTORS; TRANSGENES;

EID: 3042609692     PISSN: 14726750     EISSN: None     Source Type: Journal    
DOI: 10.1186/1472-6750-3-16     Document Type: Article

References (16)

1. Zanjani ED and Anderson WF: Prospects for in utero human gene therapy. Science 1999, 285:2084-2088.
2. Larson JE and Cohen JC: Cystic fibrosis revisited. Mol Genet Metab 2000, 71:470-477.
3. Cohen JC, Morrow SL, Cork RJ, Delcarpio JB and Larson JE: Molecular pathophysiology of cystic fibrosis based on the rescued knockout mouse model. Mol Genet Metab 1998, 64:108-118.
4. Larson JE, Delcarpio JB, Farberman MM, Morrow SL and Cohen JC: CFTR modulates lung secretory cell proliferation and differentiation. Am J Physiol Lung Cell Mol Physiol 2000, 279:L333-41.
5. Larson JE, Morrow SL, Delcarpio JB, Bohm RP, Ratterree MS, Blanchard JL and Cohen JC: Gene transfer into the fetal primate: evidence for the secretion of transgene product. Mol Ther 2000, 2:631-639.
6. Larson JE, Morrow SL, Happel L, Sharp JF and Cohen JC: Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 1997, 349:619-620.
7. Morrow SL, Larson JE, Nelson S, Sekhon HS, Ren T and Cohen JC: Modification of development by the CFTR gene in utero. Mol Genet Metab 1998, 65:203-212.
8. Sekhon HS and Larson JE: In utero gene transfer into the pulmonary epithelium. Nat Med 1995, 1:1201-1203.
9. Kotin RM, Siniscalco M, Samulski RJ, Zhu XD, Hunter L, Laughlin CA, McLaughlin S, Muzyczka N, Rocchi M and Berns KI: Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A 1990, 87:2211-2215.
10. Kotin RM, Linden RM and Berns KI: Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. Embo J 1992, 11:5071-5078.
11. Duan D, Sharma P, Yang J, Yue Y, Dudus L, Zhang Y, Fisher KJ and Engelhardt JF: Circular intermediates of recombinant adenoassociated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J Virol 1998, 72:8568-8577.
12. Monahan PE and Samulski RJ: Adeno-associated virus vectors for gene therapy: more pros than cons? Mol Med Today 2000, 6:433-440.
13. Rabinowitz JE and Samulski RJ: Building a better vector: the manipulation of AAV virions. Virology 2000, 278:301-308.
14. Boyle MP, Enke RA, Adams RJ, Guggino WB and Zeitlin PL: In utero AAV-mediated gene transfer to rabbit pulmonary epithelium. Mol Ther 2001, 4:115-121.
15. Mitchell M, Jerebtsova M, Batshaw ML, Newman K and Ye X: Longterm gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors. Gene Ther 2000, 7:1986-1992.
16. Lipshutz GS, Gruber CA, Cao Y, Hardy J, Contag CH and Gaensler KM: In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression. Mol Ther 2001, 3:284-292.