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| Verfasst von: | Kang, Hyoung Jin [VerfasserIn]  |
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| | Bartholomä, Cynthia C. [VerfasserIn] |
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| | Paruzynski, Anna [VerfasserIn] |
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| | Arens, Anne [VerfasserIn] |
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| | Kim, Sujeong [VerfasserIn] |
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| | Yu, Seung Shin [VerfasserIn] |
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| | Hong, Youngtae [VerfasserIn] |
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| | Joo, Chang-Wan [VerfasserIn] |
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| | Yoon, Nam-Kyung [VerfasserIn] |
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| | Rhim, Jung-Woo [VerfasserIn] |
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| | Kim, Joong Gon [VerfasserIn] |
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| | Kalle, Christof von [VerfasserIn] |
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| | Schmidt, Manfred [VerfasserIn] |
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| | Kim, Sunyoung [VerfasserIn] |
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| | Ahn, Hyo Seop [VerfasserIn] |
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| Titel: | Retroviral gene therapy for X-linked chronic granulomatous disease |
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| Titelzusatz: | results from phase I/II trial |
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| Verf.angabe: | Hyoung Jin Kang, Cynthia C Bartholomae, Anna Paruzynski, Anne Arens, Sujeong Kim, Seung Shin Yu, Youngtae Hong, Chang-Wan Joo, Nam-Kyung Yoon, Jung-Woo Rhim, Joong Gon Kim, Christof Von Kalle, Manfred Schmidt, Sunyoung Kim and Hyo Seop Ahn |
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| E-Jahr: | 2011 |
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| Jahr: | 30 August 2011 |
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| Umfang: | 10 S. |
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| Fussnoten: | Gesehen am 11.07.2022 |
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| Titel Quelle: | Enthalten in: Molecular therapy |
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| Ort Quelle: | Amsterdam : Elsevier, 2000 |
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| Jahr Quelle: | 2011 |
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| Band/Heft Quelle: | 19(2011), 11, Seite 2092-2101 |
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| ISSN Quelle: | 1525-0024 |
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| Abstract: | X-linked chronic granulomatous disease (CGD) is an inherited immunodeficiency caused by a defect in the gp91phox gene. In an effort to treat X-CGD, we investigated the safety and efficacy of gene therapy using a retroviral vector, MT-gp91. Two X-CGD patients received autologous CD34+ cells transduced with MT-gp91 after a conditioning regimen consisting of fludarabine and busulfan. The level of gene-marked cells was highest at day 21 (8.3 and 11.7% in peripheral blood cells) but decreased to 0.08 and 0.5%, respectively, 3 years after gene transfer. The level of functionally corrected cells, as determined by nicotinamide adenine dinucleotide phosphate (NADPH) oxidase assay, reached a peak at day 17 (6.5% patient 1 (P1) and 14.3% patient 2 (P2) of total granulocytes) and declined to 0.05% (P1) and 0.21% (P2), 3 years later. Some retroviral vectors were found to have integrated within or close to the proto-oncogenes MDS1-EVI1, PRDM16, and CCND2; however, no abnormal cell expansion or related hematological malignancy was observed. Overall, the gene transfer procedure did not produce any serious adverse effects and was able to convert a significant fraction of blood cells to biologically functional cells, albeit for a short period of time. |
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| DOI: | doi:10.1038/mt.2011.166 |
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| URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
Volltext ; Verlag: https://doi.org/10.1038/mt.2011.166 |
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| | Volltext: https://www.sciencedirect.com/science/article/pii/S1525001616328076 |
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| | DOI: https://doi.org/10.1038/mt.2011.166 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| K10plus-PPN: | 1809682126 |
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| Verknüpfungen: | → Zeitschrift |
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Retroviral gene therapy for X-linked chronic granulomatous disease / Kang, Hyoung Jin [VerfasserIn]; 30 August 2011 (Online-Ressource)
