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Behandling af monogene sygdomme ved viral transduktion af hæmatopoietiske stamceller

Forfatter(e)

Marianne Segelcke Ifversen1, Tania Nicole Masmas1, Brian Kornblit2, Klaus Rieneck3, Eva Kannik Kaastrup3, Allan Meldgaard Lund4, Anne Fischer-Nielsen5 & Andreas Glenthøj6

1) BørneUngeKlinikken, Rigshospitalet, 2) Hæmatologisk Klinik, Rigshospitalet, 3) Blodbanken, Klinisk Immunologisk Afdeling, Rigshospitalet, 4) Center for Medfødte Stofskiftesygdomme, Rigshospitalet, 5) Stamcellesektionen, Blodbanken, Rigshospitalet, 6) Videnscenter for Hæmoglobinsygdomme, Afdeling for Blodsygdomme, Herlev Hospital

Ugeskr Læger 2020;182:V06200458

Reference: 
Ugeskr Læger 2020;182:V06200458
Blad nummer: 
Treatment of monogenic disorders with viral transduced haematopoietic stem cells

Marianne Segelcke Ifversen, Tania Nicole Masmas, Brian Kornblit, Klaus Rieneck, Eva Kannik Kaastrup, Allan Meldgaard Lund, Anne Fischer-Nielsen & Andreas Glenthøj

Ugeskr Læger 2020;182:V06200458

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.

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