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Titolo:
Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII
Autore:
Kamata, Y; Okuyama, T; Kosuga, M; Ohira, A; Kanaji, A; Sasaki, K; Yamada, M; Azuma, N;
Indirizzi:
Natl Childrens Med Res Ctr, Dept Genet, Tokyo 1548509, Japan Natl Childrens Med Res Ctr Tokyo Japan 1548509 net, Tokyo 1548509, Japan Natl Childrens Hosp, Dept Ophthalmol, Tokyo 1548509, Japan Natl Childrens Hosp Tokyo Japan 1548509 Ophthalmol, Tokyo 1548509, Japan Keio Univ, Sch Med, Dept Pediat, Tokyo 1608582, Japan Keio Univ Tokyo Japan 1608582 Sch Med, Dept Pediat, Tokyo 1608582, Japan
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 4, volume: 4, anno: 2001,
pagine: 307 - 312
SICI:
1525-0016(200110)4:4<307:AGTFCC>2.0.ZU;2-6
Fonte:
ISI
Lingua:
ENG
Soggetto:
BONE-MARROW TRANSPLANTATION; ENZYME REPLACEMENT THERAPY; BETA-GLUCURONIDASE DEFICIENCY; RECOMBINANT ADENOVIRUSES; INFECTED HEPATOCYTES; IN-VIVO; MURINE; EXPRESSION; PATHOLOGY; SYSTEM;
Keywords:
mucopolysaccharidosis; adenovirus; corneal clouding;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
33
Recensione:
Indirizzi per estratti:
Indirizzo: Okuyama, T Natl Childrens Med Res Ctr, Dept Genet, Tokyo 1548509, Japan Natl Childrens Med Res Ctr Tokyo Japan 1548509 1548509, Japan
Citazione:
Y. Kamata et al., "Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII", MOL THER, 4(4), 2001, pp. 307-312

Abstract

Recent advances in systemic treatments for mucopolysaccharidosis have led to therapies that improve the multiple somatic features of this disease, but the therapeutic effect on ocular manifestations such as corneal clouding is not satisfactory. Here, we administered an adenovirus expressing human beta -glucuronidase (AxCAhGUS) into the anterior chamber or intrastromal region of the cornea in mice with mucopolysaccharidosis type VII (B6/MPSVII), and successfully treated corneal clouding of MPSVII. When we injected AxCAhGUS into the anterior chamber of the eyes, cells expressing beta -glucuronidase (GUSB) were located mainly in the trabecular meshwork as well as in all corneal regions, and subsequent pathological corrections in the cornea were achieved. Widespread transgene expression was also observed when we administered AxCAhGUS inside the cornea after lamellar keratotomy, and rapid elimination of the lysosomal storage in the corneal keratocytes occurred. Furthermore, intrastromal vector administration did not generate significant levels of anti-adenovirus neutralizing antibodies, and secondary vector administration was effective. Based on these observations, we conclude that it is worth developing a treatment strategy for corneal clouding in mucopolysaccharidosis based on direct intraocular administration of adenoviral vectors.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 10/07/20 alle ore 11:50:38