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Titolo:
Gene therapy in the CNS
Autore:
Costantini, LC; Bakowska, JC; Breakefield, XO; Isacson, O;
Indirizzi:
Massachusetts Gen Hosp E, Dept Mol Neurogenet, Charlestown, MA 02129 USA Massachusetts Gen Hosp E Charlestown MA USA 02129 arlestown, MA 02129 USA Harvard Univ, McLean Hosp, Sch Med, Neuroregenerat Lab, Belmont, MA 02178 USA Harvard Univ Belmont MA USA 02178 uroregenerat Lab, Belmont, MA 02178 USA Harvard Univ, Sch Med, Dept Neurol, Boston, MA USA Harvard Univ Boston MAUSA rd Univ, Sch Med, Dept Neurol, Boston, MA USA Harvard Univ, Sch Med, Neurosci Program, Boston, MA 02115 USA Harvard Univ Boston MA USA 02115 , Neurosci Program, Boston, MA 02115 USA
Titolo Testata:
GENE THERAPY
fascicolo: 2, volume: 7, anno: 2000,
pagine: 93 - 109
SICI:
0969-7128(200001)7:2<93:GTITC>2.0.ZU;2-M
Fonte:
ISI
Lingua:
ENG
Soggetto:
HERPES-SIMPLEX-VIRUS; RECOMBINANT ADENOASSOCIATED VIRUS; CENTRAL-NERVOUS-SYSTEM; INTERLEUKIN-1 RECEPTOR ANTAGONIST; GENETICALLY-MODIFIED FIBROBLASTS; EXPRESSING TYROSINE-HYDROXYLASE; ASTROCYTE-SPECIFIC EXPRESSION; EXPERIMENTAL BRAIN-TUMORS; AMINO-ACID DECARBOXYLASE; VIRAL CODING SEQUENCES;
Keywords:
vector; Parkinson's disease; Huntington's disease; ischemia; brain tumor; lysosomal storage disease;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
242
Recensione:
Indirizzi per estratti:
Indirizzo: Breakefield, XO Massachusetts Gen Hosp E, Dept Mol Neurogenet, 13th St,Bldg 149, Charlestown, MA 02129 USA Massachusetts Gen Hosp E 13th St,Bldg 149 Charlestown MA USA 02129
Citazione:
L.C. Costantini et al., "Gene therapy in the CNS", GENE THER, 7(2), 2000, pp. 93-109

Abstract

Gene therapy for neurological disorder is currently an experimental concept. The goals for clinical utilization are the relief of symptoms, slowing of disease progression, and correction of genetic abnormalities. Experimental studies are realizing these goals in the development of gene therapies inanimal models. Discoveries of the molecular basis of neurological disease and advances in gene transfer systems have allowed focal and global delivery of therapeutic genes for a wide variety of CNS disorders. Limitations arestill apparent, such as stability and regulation of transgene expression, and safety of both vector and expressed transgene. In addition, the brain adds several challenges not seen in peripheral gene therapy paradigms, such as post-mitotic cells, heterogeneity of cell types and circuits, and limited access. Moreover, ii is likely that several modes of gene delivery will be necessary for successful gene therapies of the CNS. Collaborative effortsbetween clinicians and basic researchers will likely yield effective gene therapy in the CNS.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 03/07/20 alle ore 01:19:08