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Titolo:
Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements
Autore:
Shen, JS; Watabe, K; Ohashi, T; Eto, Y;
Indirizzi:
Jikei Univ, Sch Med, Inst DNA Med,Dept Gene Therapy, Minato Ku, Tokyo 1058461, Japan Jikei Univ Tokyo Japan 1058461 Therapy, Minato Ku, Tokyo 1058461, Japan Jikei Univ, Sch Med, Dept Pediat, Tokyo 1058461, Japan Jikei Univ Tokyo Japan 1058461 ch Med, Dept Pediat, Tokyo 1058461, Japan Tokyo Metropolitan Inst Neurosci, Dept Neuropathol, Tokyo, Japan Tokyo Metropolitan Inst Neurosci Tokyo Japan Neuropathol, Tokyo, Japan
Titolo Testata:
GENE THERAPY
fascicolo: 14, volume: 8, anno: 2001,
pagine: 1081 - 1087
SICI:
0969-7128(200107)8:14<1081:IAORAT>2.0.ZU;2-D
Fonte:
ISI
Lingua:
ENG
Soggetto:
GLOBOID-CELL LEUKODYSTROPHY; KRABBE DISEASE; HUMAN GALACTOCEREBROSIDASE; STORAGE DISEASE; EXPRESSION; MODEL; CDNA; TRANSPLANTATION; PROTEIN; DEMYELINATION;
Keywords:
lysosomal storage disease; twitcher mouse; neonatal; gene therapy; intraventricular; recombinant adenovirus;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
27
Recensione:
Indirizzi per estratti:
Indirizzo: Ohashi, T Jikei Univ, Sch Med, Inst DNA Med,Dept Gene Therapy, Minato Ku, 3-25-8 Nishi Shinbashi, Tokyo 1058461, Japan Jikei Univ 3-25-8 Nishi Shinbashi Tokyo Japan 1058461 61, Japan
Citazione:
J.S. Shen et al., "Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements", GENE THER, 8(14), 2001, pp. 1081-1087

Abstract

Twitcher mouse is a murine model of human globoid cell leukodystrophy (Krabbe disease), which is characterized by a genetic deficiency in galactocerebrosidase (GALC) activity. The nervous system is affected early and severely by demyelination in the white matter. So far, there is no effective treatment for Krabbe disease except bone marrow transplantation (BMT), However, BMT has inherent limitations such as unavailability of donors and graft-versus-host disease. In this study, we injected recombinant adenovirus encoding GALC into the lateral ventricle of twitcher mice at postnatal day 0 (PND 0) and the therapeutic effects were evaluated. Our results showed slight, but significant improvements in motor functions, body weight and twitching and a prolonged life span. In brain, GALC activity was increased to 15% thatof normal littermates and psychosine concentration was decreased to 55% that of untreated twitcher mice at PND 15. The number of PAS-positive globoidcells in brain stem was also reduced significantly at PND 35. In contrast,when adenoviruses were injected to the twitcher mice at PND 15, almost no improvements were observed. These results demonstrate that the timing of treatment may be of great importance in Krabbe disease.

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Documento generato il 23/09/20 alle ore 09:41:45