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Titolo:
Gene therapy: Promises and problems
Autore:
Pfeifer, A; Verma, IM;
Indirizzi:
Salk Inst Biol Studies, La Jolla, CA 92037 USA Salk Inst Biol Studies La Jolla CA USA 92037 dies, La Jolla, CA 92037 USA
Titolo Testata:
ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS
, volume: 2, anno: 2001,
pagine: 177 - 211
SICI:
1527-8204(2001)2:<177:GTPAP>2.0.ZU;2-Z
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; VESICULAR STOMATITIS-VIRUS; HUMAN ADENOVIRUS TYPE-5; PACKAGING CELL-LINES; POSTTRANSCRIPTIONAL REGULATORY ELEMENT; PSEUDOTYPED RETROVIRAL VECTORS; INACTIVATING LENTIVIRUS VECTOR; HUMAN HEMATOPOIETIC-CELLS; NONDIVIDING HUMAN-CELLS; CRE-MEDIATED EXCISION;
Keywords:
gene transfer; viral vectors; gene therapy trials;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
190
Recensione:
Indirizzi per estratti:
Indirizzo: Pfeifer, A Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies 10010 N Torrey Pines Rd La Jolla CA USA 92037
Citazione:
A. Pfeifer e I.M. Verma, "Gene therapy: Promises and problems", ANN REV GEN, 2, 2001, pp. 177-211

Abstract

Gene therapy can be broadly defined as the transfer of genetic material tocure a disease or at least to improve the clinical status of a patient. One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells. Based on the nature of the viral genome, these gene therapy vectors can be divided into RNA and DNA viral vectors. The majority of RNA virus-based vectors have been derived from simple retroviruses like murine leukemia virus. A major shortcoming of these vectors is that they are not able to transduce nondividing cells. This problem may be overcome by the use of novel retroviral vectors derived from lentiviruses, such as human immunodeficiency virus (HIV). The most commonly used DNA virus vectors are based on adenoviruses and adeno-associated viruses. Although the available vector systems are able to deliver genes in vivo into cells, the ideal delivery vehicle has notbeen found. Thus, the present viral vectors should be used only with greatcaution in human beings and further progress in vector development is necessary.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 25/02/20 alle ore 14:24:29