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Titolo:
Feasibility of gene therapy for late neuronal ceroid lipofuscinosis
Autore:
Sondhi, D; Hackett, NR; Apblett, RL; Kaminsky, SM; Pergolizzi, RG; Crystal, RG;
Indirizzi:
Cornell Univ, Weill Med Coll, Inst Med Genet, New York, NY 10021 USA Cornell Univ New York NY USA 10021 Inst Med Genet, New York, NY 10021 USA Cornell Univ, Weill Med Coll, Belfer Gene Therapy Core Facil, New York, NY10021 USA Cornell Univ New York NY USA 10021 rapy Core Facil, New York, NY10021 USA
Titolo Testata:
ARCHIVES OF NEUROLOGY
fascicolo: 11, volume: 58, anno: 2001,
pagine: 1793 - 1798
SICI:
0003-9942(200111)58:11<1793:FOGTFL>2.0.ZU;2-U
Fonte:
ISI
Lingua:
ENG
Soggetto:
MUCOPOLYSACCHARIDOSIS TYPE-VII; BONE-MARROW TRANSPLANTATION; CENTRAL-NERVOUS-SYSTEM; RECOMBINANT ADENOASSOCIATED VIRUS; HUMAN BETA-GLUCURONIDASE; LYSOSOMAL STORAGE; RETINAL DEGENERATION; NONDIVIDING CELLS; MOUSE-BRAIN; AAV VECTOR;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Life Sciences
Citazioni:
41
Recensione:
Indirizzi per estratti:
Indirizzo: Crystal, RG Cornell Univ, Weill Med Coll, Inst Med Genet, 515 E 70st St,Suite 1000, New York, NY 10021 USA Cornell Univ 515 E 70st St,Suite 1000 New York NY USA 10021 SA
Citazione:
D. Sondhi et al., "Feasibility of gene therapy for late neuronal ceroid lipofuscinosis", ARCH NEUROL, 58(11), 2001, pp. 1793-1798

Abstract

Late infantile neuronal ceroid lipofuscinosis is a progressive childhood neurodegenerative disorder characterized by intracellular accumulation of autofluorescent material resembling lipofuscin in neuronal cells. This reportsummarizes the new therapies under consideration for late infantile neuronal ceroid lipofuscinosis, with a focus on strategies for in vivo gene therapy for the retinal and central nervous system manifestations of the disease.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 05/06/20 alle ore 14:52:55