DK1950307T3 - Genoverførsel til luftvejsepitelstamcelle ved anvendelse af en lentivirusvektor, som er pseudo-typificeret med RNA-virus-spike-protein - Google Patents

Genoverførsel til luftvejsepitelstamcelle ved anvendelse af en lentivirusvektor, som er pseudo-typificeret med RNA-virus-spike-protein Download PDF

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DK1950307T3
DK1950307T3 DK06822478.1T DK06822478T DK1950307T3 DK 1950307 T3 DK1950307 T3 DK 1950307T3 DK 06822478 T DK06822478 T DK 06822478T DK 1950307 T3 DK1950307 T3 DK 1950307T3
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vector
virus
gene
vectors
cells
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Eric W Alton
Uta Griesenbach
Katsuyuki C O Id Pharma Co Ltd Mitomo
Mamoru C O Id Pharma Co Ltd Hasegawa
Makoto C O Id Pharma Co Ltd Inoue
Hitoshi C O Id Pharma Co Ltd Iwasaki
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Id Pharma Co Ltd
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Claims (5)

1. Rekombinant SIV-vektor til anvendelse ved behandling af cystisk fibrose ved indføring af et gen i en luftvejsepitelstamcelle, som ikke er blevet udsat for en forbehandling eller en prækonditionering, hvor vektoren (i) er pseudo-typificeret med spike-proteiner af Sendai virus-envelope-glykoproteinerne F og HN, og (ii) omfatter et fremmed gen i en eksprimerbar tilstand, og desuden hvor vektoren muliggør integreringen af det fremmede gen i stamcellen, således at der tilvejebringes en langvarig ekspression af det fremmede gen på mindst 360 dage.
2. Anvendelse af en rekombinant SIV-vektor ved fremstilling af et medikament til behandling af en cystisk fibrose, hvor: (a) vektoren er pseudo-typificeret med spike-proteiner af Sendai virus-envelope-glykoproteinerne F og FIN og omfatter et fremmed gen i en eksprimerbar tilstand, og hvor vektoren muliggør integreringen af det fremmede gen i en luftvejsepitelstamcelle, således at der tilvejebringes en langvarig ekspression af det fremmede gen på mindst 360 dage; og (b) medikamentet er til indgivelse i luftvejsepitelstamceller.
3. Vektor ifølge krav 1 eller anvendelse ifølge krav 2, hvor den rekombinante SIV-vektor stammer fra en agm-stamme.
4. Vektor eller anvendelse ifølge krav 3, hvor den rekombinante SIV-vektor er en selvinaktiverende vektor, hvor promotorsekvensen for 3' LTR U3-regionen er elimineret og/eller substitueret med anden promotersekvens.
5. Vektor ifølge krav 1, 3 eller 4 eller anvendelse ifølge et af kravene 2 til 4, hvor det fremmede gen er et gen, der koder for: (a) en iboende eller erhvervet dysfunktionel cystisk fibrose (CF)-forårsagende faktor; (b) et iboende eller erhvervet dysfunktionelt CFTR-protein; (c) et protein med en terapeutisk virkning på cystisk fibrose; (d) et CFTR-protein, som er nødvendig for behandlingen af cystisk fibrose; eller (e) et CFTR-protein, hvor ekspression af CFTR-proteinet supplerer et CFTR-gen, der mangler.
DK06822478.1T 2005-10-28 2006-10-27 Genoverførsel til luftvejsepitelstamcelle ved anvendelse af en lentivirusvektor, som er pseudo-typificeret med RNA-virus-spike-protein DK1950307T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2005313971 2005-10-28
PCT/JP2006/321517 WO2007049752A1 (ja) 2005-10-28 2006-10-27 Rnaウイルスまたはdnaウイルスのスパイクタンパク質でシュードタイプ化したレンチウイルスベクターを用いた気道上皮幹細胞への遺伝子導入

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DK1950307T3 true DK1950307T3 (da) 2016-02-15

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US (4) US20090162320A1 (da)
EP (2) EP1950307B8 (da)
JP (3) JPWO2007049752A1 (da)
KR (1) KR20080068098A (da)
CN (1) CN101351557A (da)
CA (1) CA2627485C (da)
CY (1) CY1117307T1 (da)
DK (1) DK1950307T3 (da)
ES (2) ES2561060T3 (da)
HU (1) HUE028512T2 (da)
PT (1) PT1950307E (da)
SI (1) SI1950307T1 (da)
WO (1) WO2007049752A1 (da)

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JP5776662B2 (ja) 2015-09-09
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US20190225986A1 (en) 2019-07-25
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US20160281109A1 (en) 2016-09-29
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