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Treatment of monogenic disorders with viral transduced haematopoietic stem cells

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

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@article{15fe485dcff247bb8f171a88c7faf374,
title = "Treatment of monogenic disorders with viral transduced haematopoietic stem cells",
abstract = "Infusion of ex vivo transduced haematopoietic stem cells (HSC) has emerged as a promising new treatment of certain monogenetic disorders. Since early clinical studies on patients with severe combined immune deficiency were halted due to de novo leukaemia, the technology has matured. Thus, treatment of transfusion-dependent thalassaemia and adenosine deaminase deficient severe combined immunodeficiency by using lentiviral vectors for gene correction of autologous HSC can induce expression of the deficient protein and thus potentially cure the patients. The review summarises recent advances allowing for clinical implementation of the treatment in Denmark.",
author = "Ifversen, {Marianne Segelcke} and Masmas, {Tania Nicole} and Brian Kornblit and Klaus Rieneck and Kaastrup, {Eva Kannik} and Lund, {Allan Meldgaard} and Anne Fischer-Nielsen and Andreas Glenth{\o}j",
year = "2020",
month = nov,
day = "9",
language = "Dansk",
volume = "182",
journal = "Ugeskrift for Laeger",
issn = "0041-5782",
publisher = "Almindelige Danske Laegeforening",
number = "46",

}

RIS

TY - JOUR

T1 - Treatment of monogenic disorders with viral transduced haematopoietic stem cells

AU - Ifversen, Marianne Segelcke

AU - Masmas, Tania Nicole

AU - Kornblit, Brian

AU - Rieneck, Klaus

AU - Kaastrup, Eva Kannik

AU - Lund, Allan Meldgaard

AU - Fischer-Nielsen, Anne

AU - Glenthøj, Andreas

PY - 2020/11/9

Y1 - 2020/11/9

N2 - Infusion of ex vivo transduced haematopoietic stem cells (HSC) has emerged as a promising new treatment of certain monogenetic disorders. Since early clinical studies on patients with severe combined immune deficiency were halted due to de novo leukaemia, the technology has matured. Thus, treatment of transfusion-dependent thalassaemia and adenosine deaminase deficient severe combined immunodeficiency by using lentiviral vectors for gene correction of autologous HSC can induce expression of the deficient protein and thus potentially cure the patients. The review summarises recent advances allowing for clinical implementation of the treatment in Denmark.

AB - Infusion of ex vivo transduced haematopoietic stem cells (HSC) has emerged as a promising new treatment of certain monogenetic disorders. Since early clinical studies on patients with severe combined immune deficiency were halted due to de novo leukaemia, the technology has matured. Thus, treatment of transfusion-dependent thalassaemia and adenosine deaminase deficient severe combined immunodeficiency by using lentiviral vectors for gene correction of autologous HSC can induce expression of the deficient protein and thus potentially cure the patients. The review summarises recent advances allowing for clinical implementation of the treatment in Denmark.

M3 - Tidsskriftartikel

C2 - 33215591

VL - 182

JO - Ugeskrift for Laeger

JF - Ugeskrift for Laeger

SN - 0041-5782

IS - 46

ER -

ID: 61660916