DK2802662T3 - Anvendelse af pseudotype-lentiviruspartikler til målrettet transduktion in vitro af udifferentierede pluripotente humane embryonale stamceller og inducerede pluripotente stamceller - Google Patents

Anvendelse af pseudotype-lentiviruspartikler til målrettet transduktion in vitro af udifferentierede pluripotente humane embryonale stamceller og inducerede pluripotente stamceller Download PDF

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DK2802662T3
DK2802662T3 DK13700169.9T DK13700169T DK2802662T3 DK 2802662 T3 DK2802662 T3 DK 2802662T3 DK 13700169 T DK13700169 T DK 13700169T DK 2802662 T3 DK2802662 T3 DK 2802662T3
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Christian Buchholz
Irene Schneider
Gerald Schumann
Sabine Jung-Klawitter
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Paul Ehrlich Inst Bundesamt Fuer Sera Und Impfstoffe
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    • C07K16/2878—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
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Claims (10)

1. Anvendelse af en pseudotype-lentivirusvektorpartikel i målrettet transduktion in-vitro af undifferentierede pluripotente humane embryonale stamceller (hESC) og inducerede pluripotente stamceller (iPSC), hvilken vektor omfatter et morbillivirusfusion (F)-protein og et muteret hemagglutinin (H)-protein fra mæslingevirussen (MeV) eller Edmonton-mæslingevirusstammen (MeVEdm), hvor de cytoplasmatiske andele af F- og H-proteinet er afkortet, og det afkortede F-protein er FcA24 (24 aminosyrer fjernet fra C-terminalen) eller FcA30 (30 aminosyrer fjernet fra C-terminalen), og det muterede og afkortede H-protein er udvalgt fra gruppen bestående af HcA14 (aminosyre 2-15 fjernet), HcA15 (aminosyre 2-16 fjernet), HcA16 (aminosyre 2-17 fjernet), HcA17 (aminosyre 2-18 fjernet), HcA18 (aminosyre 2-19 fjernet), HcA19 (aminosyre 2-20 fjernet), HcA20 (aminosyre 2-21 fjernet), HcA21+A (aminosyre 2-22 erstattet med én alanin) og HcA24+4A (aminosyre 2-25 erstattet med fire alaniner), fortrinsvis HcA18, HcA19 og HcA24+4A, hvor de aminosyrer, der er nødvendige for receptorgenkendelse i H-proteinet er muterede, så det ikke interagerer med CD46, SLAM og/eller nectin-4, og hvor H-proteinet endvidere har et enkeltkædet antistof rettet mod CD30, EpCAM (CD326), CD9, Thy-1 (CD90), SSEA-3, SSEA-4, TRA-1-60 eller TRA-1-81 på dets ectodomæne.
2. Anvendelse ifølge krav 1, hvor mutationen i H-proteinet er: - mindst én punktmutation med en anden aminosyre ved resterne, der er udvalgt fra gruppen bestående af V451, Y529, Y481, F431, V451, Y452, A527, P486,1487, A428, L464, R533, G546, S548 og F549 med alanin, leucin, serin, methionin eller glutamin; og/eller - en punktmutation af alle fem på hinanden følgende rester 473 til 477 med alanin; og/eller - mindst én punktmutation med en anden aminosyre på resterne, der er udvalgt fra gruppen bestående af 1194, D530, Y553, T531, P554, F552, D505 og D507; og/eller - mindst én punktmutation med en anden aminosyre ved resterne, der er udvalgt fra gruppen bestående af Y543 og P497.
3. Anvendelse ifølge et hvilket som helst af kravene 1 til 2, hvor scFv'en er rettet mod CD30 med den optimerede aminosyresekvens som vist i fig. 5.
4. Anvendelse ifølge et hvilket som helst af kravene 1 til 3, hvor pseudotype-lentivirusvektorpartiklen er afledt af en lentivirus, der er udvalgt fra gruppen bestående af HIV-1, HIV-2, SIVmac, SIVPbj, SIVagm, FIV og EIAV, fortrinsvis HIV-1.
5. Anvendelse ifølge krav 4, hvor pseudotype-lentivirusvektorpartiklen ikke omfatter den genetiske information fra generne gag, env og/eller pol og/eller fra andre ikke-essentielle gener, der er udvalgt fra gruppen bestående af tat, vif, vpr; vpu og nef.
6. Anvendelse ifølge krav 5, hvor generne tilvejebringes i trans ved hjælp af en pakkecellelinje, som er udvalgt fra gruppen bestående af humane embryonale 293-nyreceller, der indeholder SV40 Large T-antigenet (HEK-293T eller 293T), human sarkomcellelinje FIT-1080 (CCL-121), lymfoblastlignende cellelinje Raji (CCL-86), glioblastomastrocytom-epithellignende cellelinje U87-MG (HTB-14) og T-lymfomcellelinje HuT78 (TIB-161), mere fortrinsvis HEK-293T.
7. Anvendelse ifølge et hvilket som helst af kravene 1 til 6, hvor mængden af det afkortede F-protein, der er til stede i vektorpartiklen, er højere end mængden af muteret og afkortet H-protein, der er til stede i vektorpartiklen, fortrinsvis 10-100 %, 100-250 %, 250-500 %, 500-750 %, 750-1000 % eller mere end 1000 % højere og, fortrinsvis, 700 % højere end mængden af muteret og afkortet H-protein.
8. Anvendelse ifølge et hvilket som helst af kravene 1 til 7, der endvidere omfatter en psi-positiv RNA-ekspressionsvektor, hvor den psi-positive RNA-ekspressionsvektor omfatter mindst ét selekterbart markørgen.
9. Anvendelse ifølge krav 8, hvor det selekterbare markørgen er udvalgt fra gruppen bestående af et gen, der koder for GFP, et gen, der koder for eGFP, et gen, der koder for et apoptosefremkaldende protein, et gen, der koder for et cytotoksisk protein, TNF-α-gen, p53-gen, en interfererende RNA, et interferon-gen, herpes virus-thymidinkinase-genet, et gen, der koder for et immunstimulerende protein, og et gen, der koder for et terapeutisk protein.
10. Anvendelse ifølge et hvilket som helst af kravene 1-9, hvor iPSC'erne er udvalgt fra gruppen bestående af hFFBiPS SB4 og HFFBiPS SB5 eller er genereret fra patienter med adenosin-deaminasedefekt-relateret svær kombineret immundefekt (ADA-SCID), Duchenne (DMD) og Becker muskeldystrofi (BMD), Parkinsons sygdom (PD) eller juvenil type 1-diabetes mellitus (JDM).
DK13700169.9T 2012-01-11 2013-01-10 Anvendelse af pseudotype-lentiviruspartikler til målrettet transduktion in vitro af udifferentierede pluripotente humane embryonale stamceller og inducerede pluripotente stamceller DK2802662T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP12000142.5A EP2615176A1 (en) 2012-01-11 2012-01-11 Novel pseudotyped lentiviral particles and their use in the in vitro targeted transduction of undifferentiated pluripotent human embryonic stem cells and induced pluripotent stem cells
PCT/EP2013/050426 WO2013104728A1 (en) 2012-01-11 2013-01-10 Novel pseudotyped lentiviral particles and their use in the in vitro targeted transduction of undifferentiated pluripotent human embryonic stem cells and induced pluripotent stem cells

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US (1) US20150291979A1 (da)
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WO (1) WO2013104728A1 (da)

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CN107805628A (zh) * 2017-10-26 2018-03-16 安徽农业大学 一种稳定表达小反刍兽疫病毒受体Nectin‑4的细胞系及其构建方法
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CN109266614A (zh) * 2018-08-17 2019-01-25 中国农业科学院兰州兽医研究所 一种基于小反刍兽疫病毒受体的细胞BHK/slam
CN109266615A (zh) * 2018-08-17 2019-01-25 中国农业科学院兰州兽医研究所 一种基于小反刍兽疫病毒受体的细胞BHK/slam/v
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EP4320242A1 (en) 2021-04-08 2024-02-14 Sana Biotechnology, Inc. Cd8-specific antibody constructs and compositions thereof
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WO2023196742A1 (en) * 2022-04-08 2023-10-12 Fred Hutchinson Cancer Center Anti-cd90 antibodies, binding fragments, and uses thereof

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EP2802662B1 (en) 2018-12-26
WO2013104728A1 (en) 2013-07-18
ES2717274T3 (es) 2019-06-20
US20150291979A1 (en) 2015-10-15
EP2802662B8 (en) 2019-02-20
EP2615176A1 (en) 2013-07-17

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