Stability and expression of human MDR1 in retroviral vectors for gene therapy.

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00023736%3A_____%2F02%3A00000098" target="_blank" >RIV/00023736:_____/02:00000098 - isvavai.cz</a>

Výsledek na webu

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DOI - Digital Object Identifier

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Jazyk výsledku

angličtina

Název v původním jazyce

Stability and expression of human MDR1 in retroviral vectors for gene therapy.

Popis výsledku v původním jazyce

The human multidrug resistance 1 (hMDR1) gene encodes a membrane-located protein (P-gp), which is capable to pump xenobiotics out of cells. This property makes hMDR1 potentially useful for gene therapy, since its overexpression can protect genetically modified hematopoietic cells against the effects of cytotoxic agents. However, ectopic expression of hMDR1 mRNA from retroviral vectors is compromised by an aberrant splicing which results in a production of a non-functional protein. Here we report that 1)our construct (QQ-SA) improved the functional properties of MDR1 compared with the wild type, although the aberrant splicing was not fully eliminated. 2) The other mutations tested resulted in the elimination of the aberrant splicing, however, they hada negative impact on the P-gp function. 3) The use of human packaging cells GP2-293 greatly reduced the frequency of MDR1 aberrant splicing in retroviral vectors and seems to be a valuable tool for generating retroviruses containing MDR1

Název v anglickém jazyce

Stability and expression of human MDR1 in retroviral vectors for gene therapy.

Popis výsledku anglicky

The human multidrug resistance 1 (hMDR1) gene encodes a membrane-located protein (P-gp), which is capable to pump xenobiotics out of cells. This property makes hMDR1 potentially useful for gene therapy, since its overexpression can protect genetically modified hematopoietic cells against the effects of cytotoxic agents. However, ectopic expression of hMDR1 mRNA from retroviral vectors is compromised by an aberrant splicing which results in a production of a non-functional protein. Here we report that 1)our construct (QQ-SA) improved the functional properties of MDR1 compared with the wild type, although the aberrant splicing was not fully eliminated. 2) The other mutations tested resulted in the elimination of the aberrant splicing, however, they hada negative impact on the P-gp function. 3) The use of human packaging cells GP2-293 greatly reduced the frequency of MDR1 aberrant splicing in retroviral vectors and seems to be a valuable tool for generating retroviruses containing MDR1

Druh

D - Stať ve sborníku

CEP obor

FD - Onkologie a hematologie

OECD FORD obor

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Projekt

<a href="/cs/project/NE6689" target="_blank" >NE6689: Vrozené defekty hematopoesy</a><br>

Návaznosti

P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)

Rok uplatnění

2002

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název statě ve sborníku

XIII. český a slovenský hematologický a transfuziologický kongres s mezinárodní účastí.

ISBN

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ISSN

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e-ISSN

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Počet stran výsledku

1

Strana od-do

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Název nakladatele

Agentura Carolina

Místo vydání

Praha

Místo konání akce

Praha

Datum konání akce

1. 1. 2002

Typ akce podle státní příslušnosti

EUR - Evropská akce

Kód UT WoS článku

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