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Titolo:
In vivo treatment of hemophilia A and mucopolysaccharidosis type VII usingnonprimate lentiviral vectors
Autore:
Stein, CS; Kang, Y; Sauter, SL; Townsend, K; Staber, P; Derksen, TA; Martins, I; Qian, J; Davidson, BL; McCray, PB;
Indirizzi:
Univ Iowa, Coll Med, Dept Internal Med, Program Gene Therapy, Iowa City, IA 52242 USA Univ Iowa Iowa City IA USA 52242 am Gene Therapy, Iowa City, IA 52242 USA Univ Iowa, Coll Med, Dept Neurol, Iowa City, IA 52242 USA Univ Iowa Iowa City IA USA 52242 ed, Dept Neurol, Iowa City, IA 52242 USA Univ Iowa, Coll Med, Dept Physiol & Biophys, Iowa City, IA 52242 USA Univ Iowa Iowa City IA USA 52242 ysiol & Biophys, Iowa City, IA 52242 USA Univ Iowa, Coll Med, Dept Pediat, Iowa City, IA 52242 USA Univ Iowa Iowa City IA USA 52242 ed, Dept Pediat, Iowa City, IA 52242 USA Chiron Technol, Ctr Gene Therapy, San Diego, CA 92121 USA Chiron Technol San Diego CA USA 92121 ne Therapy, San Diego, CA 92121 USA Univ Minnesota, Dept Biochem, St Paul, MN 55108 USA Univ Minnesota St Paul MN USA 55108 , Dept Biochem, St Paul, MN 55108 USA
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 6, volume: 3, anno: 2001,
pagine: 850 - 856
SICI:
1525-0016(200106)3:6<850:IVTOHA>2.0.ZU;2-L
Fonte:
ISI
Lingua:
ENG
Soggetto:
MEDIATED GENE-TRANSFER; IMMUNODEFICIENCY VIRUS VECTORS; CENTRAL-NERVOUS-SYSTEM; BETA-GLUCURONIDASE; RETROVIRAL VECTOR; LYSOSOMAL STORAGE; IN-VIVO; EFFICIENT TRANSDUCTION; SKIN FIBROBLASTS; LIVER;
Keywords:
hemophilia A; gene therapy; lentiviral vector; mucopolysaccharidosis; beta-glucuronidase;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
32
Recensione:
Indirizzi per estratti:
Indirizzo: McCray, PB Univ Iowa, Coll Med, Dept Internal Med, Program Gene Therapy, Iowa City, IA 52242 USA Univ Iowa Iowa City IA USA 52242 rapy, Iowa City, IA 52242 USA
Citazione:
C.S. Stein et al., "In vivo treatment of hemophilia A and mucopolysaccharidosis type VII usingnonprimate lentiviral vectors", MOL THER, 3(6), 2001, pp. 850-856

Abstract

Gene therapy holds great promise for the treatment of a variety of inherited diseases, including hemophilia A and mucopolysaccharidosis type VII (MPSVII). In both these disorders, subnormal levels of replacement protein have therapeutic effects. Thus we hypothesized that transduction of a small proportion of cells by feline immunodeficiency virus (FIV)-based lentiviral vectors might provide sufficient levels of transgene expression for phenotypic correction. We intravenously injected replication-deficient FIV-based vectors encoding either human factor VIII or human beta -glucuronidase into factor VIII-deficient or beta -glucuronidase-deficient mice, respectively. This route of delivery targeted multiple organs, with the liver as the primary transduction site. In the hemophilia A mice, factor VIII expression persisted for the duration of the experiments (approximately 5 months), and recipient mice survived an otherwise lethal bleeding episode (tall-clipping). In mucopolysaccharidosis type VII mice, substantial beta -glucuronidase activity was detected in several tissues and corresponded with marked reduction of lysosomal storage in liver and spleen. These findings indicate that gene transfer with FIV-based lentiviral vectors can permanently introduce transgenes into a sufficient number of hepatocytes for long-term therapeutic effect and suggest potential clinical value of FIV-based lentiviral vectors for treatment of hemophilia A and MPS VII.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 28/01/20 alle ore 15:36:02