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    STABLE PSEUDOTYPED LENTIVIRAL PARTICLES AND USES THEREOF
    113.
    发明公开
    STABLE PSEUDOTYPED LENTIVIRAL PARTICLES AND USES THEREOF 审中-公开

    公开(公告)号：US20230203537A1

    公开(公告)日：2023-06-29

    申请号：US17929816

    申请日：2022-09-06

    Applicant: UNIVERSITE D'EVRY VAL D'ESSONNE ,  GENETHON ,  INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÈDICAL)

    Inventor： Anne GALY

    IPC: C12N15/86 ,  A61K35/76 ,  C07K14/005 ,  C12N5/0781 ,  A61K35/17

    CPC classification number: C12N15/86 ,  A61K35/76 ,  C07K14/005 ,  C12N5/0635 ,  A61K35/17 ,  A61K48/00

    Abstract: The present invention relates to a method for obtaining stable pseudotyped lentiviral particles including a heterologous gene of interest, comprising the following steps:



    a) transfecting at least one plasmid in appropriate cell lines, wherein said at least one plasmid comprises the gene of interest, the rev, gag and pol genes, and a sequence coding for an ERV syncytin, wherein the rev, gag and pol genes are retroviral genes;
    b) incubating the transfected cells obtained in a), so that they produce the stable pseudotyped lentiviral particles in the supernatant; and
    c) harvesting and concentrating the stable lentiviral particles obtained in b).




    The present invention also relates to a method to transduce immune cells using lentiviral vectors pseudotyped with an ERV syncytin glycoprotein. The method can be performed on non-stimulated blood cells or on cells stimulated briefly with IL7, and the cells can be expanded.
    The stable pseudotyped lentiviral particles obtained are particularly useful in gene therapy.

    INDUCING IMMUNE TOLERANCE BY rAAV VECTORS
    119.
    发明申请
    INDUCING IMMUNE TOLERANCE BY rAAV VECTORS 有权

    公开(公告)号：US20220213505A1

    公开(公告)日：2022-07-07

    申请号：US17610840

    申请日：2020-05-15

    Applicant: UNIVERSITÉ DE PARIS ,  GENETHON ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE ,  INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

    Inventor： Federico MINGOZZI ,  Jean DAVOUST ,  Laurent BARTOLO

    IPC: C12N15/86 ,  A61P21/00 ,  A61P37/06 ,  A61K48/00

    Abstract: A combination of two recombinant adeno-associated viral (rAAV) vectors comprising in the first a capsid and a cassette comprising a 5′ ITR sequence, a liver-specific promotor, a nucleic acid sequence coding for a transgene of interest useful to be tolerated by the immune system and a poly A chain and in the second a capsid and a cassette comprising a 5′ ITR sequence, a promotor specific for a tissue of interest, a nucleic acid sequence corresponding to the nucleic acid sequence inserted into the cassette of the first rAAV vector, a transmembrane sequence, a poly A chain, wherein the nucleic acid sequences is administered towards to the tissue of interest, is disclosed. Said combination can be used in inducing an immune tolerance to a protein product encoded, and so be used as a drug in a subject, particularly for treating muscular dystrophies. Pharmaceutical compositions, kits and methods are also disclosed.

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