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Titolo:
Stem cell transplantation and gene therapy in utero
Autore:
Surbek, DV; Schatt, S; Holzgreve, W;
Indirizzi:
Univ Basel, Kantonsspital, Frauenklin, CH-4031 Basel, Switzerland Univ Basel Basel Switzerland CH-4031 uenklin, CH-4031 Basel, Switzerland
Titolo Testata:
INFUSION THERAPY AND TRANSFUSION MEDICINE-INFUSIONSTHERAPIE UND TRANSFUSIONSMEDIZIN
fascicolo: 3, volume: 28, anno: 2001,
pagine: 150 - 158
SICI:
1424-5485(200105)28:3<150:SCTAGT>2.0.ZU;2-7
Fonte:
ISI
Lingua:
ENG
Soggetto:
SEVERE COMBINED IMMUNODEFICIENCY; UMBILICAL-CORD BLOOD; FETAL HEMATOPOIETIC-CELLS; BONE-MARROW TRANSPLANTATION; L-IDURONIDASE DEFICIENCY; LONG-TERM ENGRAFTMENT; TRANSFER IN-UTERO; PROGENITOR CELLS; NOD/SCID MICE; MULTILINEAGE ENGRAFTMENT;
Keywords:
in utero transplantation; hematopoietic stem cells; fetal gene therapy;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
110
Recensione:
Indirizzi per estratti:
Indirizzo: Holzgreve, W Univ Basel, Kantonsspital, Frauenklin, Schanzenstr 46, CH-4031 Basel, Switzerland Univ Basel Schanzenstr 46 Basel Switzerland CH-4031 tzerland
Citazione:
D.V. Surbek et al., "Stem cell transplantation and gene therapy in utero", INFUS THER, 28(3), 2001, pp. 150-158

Abstract

Background: Allogeneic hematopoietic stem cell transplantation in utero has been successfully used for the prenatal treatment of severe combined immunodeficiency syndrome. However, this therapy has not been successful in thetreatment of other conditions in which the fetus is immunologically competent. Material and Methods: We reviewed the currently explored strategies toovercome these problems, including prenatal gene therapy using ex vivo transduced autologous hematopoietic cells or direct gene targeting in utero. Results: Some of the strategies such as stromal cell co-transplantation havebeen shown to be successful in preclinical studies. Similarly, prenatal gene transfer has been shown to be feasible in the fetal sheep model; however, safety concerns regarding transduction of fetal germ cells or maternal cells remain. Conclusion: Progress is being made in the exploration of new modalities of in utero transplantation although the procedure remains experimental and long-term clinical efficacy needs to be proven. In utero gene therapy seems feasible, but more animal studies are needed in order to assess its safety.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 01/04/20 alle ore 23:21:53