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Titolo:
Gene therapy for lysosomal storage disorders
Autore:
Novelli, EM; Barranger, JA;
Indirizzi:
Univ Pittsburgh, Dept Human Genet, Pittsburgh, PA 15260 USA Univ Pittsburgh Pittsburgh PA USA 15260 n Genet, Pittsburgh, PA 15260 USA
Titolo Testata:
EXPERT OPINION ON BIOLOGICAL THERAPY
fascicolo: 5, volume: 1, anno: 2001,
pagine: 857 - 867
SICI:
1471-2598(200109)1:5<857:GTFLSD>2.0.ZU;2-L
Fonte:
ISI
Lingua:
ENG
Soggetto:
MUCOPOLYSACCHARIDOSIS TYPE-VII; BONE-MARROW TRANSPLANTATION; ACID-ALPHA-GLUCOSIDASE; RETROVIRUS-MEDIATED TRANSFER; LONG-TERM EXPRESSION; DISEASE TYPE-II; MICROENCAPSULATED RECOMBINANT CELLS; HUMAN HEMATOPOIETIC-CELLS; MESENCHYMAL STEM-CELLS; A-DEFICIENT MICE;
Keywords:
animal models; BMT; enzyme replacement; gene transfer; lysosomal storage disorders; therapy;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
104
Recensione:
Indirizzi per estratti:
Indirizzo: Barranger, JA E1650 Biomed Sci Tower, Pittsburgh, PA 15261 USA E1650 Biomed Sci Tower Pittsburgh PA USA 15261 PA 15261 USA
Citazione:
E.M. Novelli e J.A. Barranger, "Gene therapy for lysosomal storage disorders", EXPERT OP B, 1(5), 2001, pp. 857-867

Abstract

The lysosomal storage disorders (LSD) are monogenic inborn errors of metabolism with heterogeneous pathophysiology and clinical manifestations. In the last decades, these disorders have been models for the development of molecular and cellular therapies for inherited metabolic diseases. Studies in preclinical in vitro systems and animal models have allowed the successful development of bone marrow transplantation (BMT) and enzyme replacement therapy (ERT) as therapeutic options for several LSDs. However, BMT is limitedby poor donor availability and high morbidity and mortality, and ERT is not a life-long cure. Moreover, the neuropathology present in many LSDs responded poorly, if at all, to these treatments. Therefore, gene therapy is an attractive therapeutic alternative. Gene therapy strategies for LSDs have employed ex vivo gene transduction of cellular targets with subsequent transplantation of the enzymatically corrected cells, or direct in vivo deliveryof the viral vectors. Oncoretroviral vectors and more recently adeno associated vectors (AAV) and lentiviral vectors have been extensively tested, with some success. This review summarises the main gene therapy strategies which have been employed or are under development for both non-neurological and neuronopathic LSDs. Some of the in vitro and in vivo preclinical studiespresented herein have provided the rationale for a gene therapy clinical trial for Gaucher disease Type I.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 19/02/20 alle ore 14:36:22