US 6,969,528 B1Granted

Phosphorus-containing dendrimers as transfection agents

AssigneeCentre National de la Recherche Scientifique (CNRS)

InventorsJean-Pierre Majoral, Bernard Meunier, Anne-Marie Caminade, Christophe Loup, Maria-Antonietta Zanta-Boussif

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Abstract

The invention concerns phosphorus-containing dendrimers and their uses, as gene transfection agents, in vitro and in vivo, including in the treatment of human and animal diseases. These agents or vectors are in particular suited for delivering to appropriate target cells, nucleic acid sequences of interest.

Claims9

Dependent claims

A polycationic phosphorus-containing dendrimer, which consists:

of a central layer in the form of a central core P0 comprising 2 to 8 functionalized groups,
of n intermediate layers, which may be identical or different, each one of said intermediate layers consisting of units P1 corresponding to formula II:
in which;
L is an oxygen, phosphorus or sulfur atom,
M represents one of the following groups:
an aromatic group di-, tri- or tetra-substituted with alkyl, alkoxy or unsaturated C1–C12 olefinic, azoic or acetylenic groups, unsubstituted or substituted with at least one phosphorous, oxygen, nitrogen, sulfur or halogen atom, or
an alkyl or alkoxy group comprising several substituents as defined when M is an aromatic group,
R1 and R2, which may be identical or different, represent a hydrogen atom or one of the following groups:
alkyl, alkoxy, or aryl, unsubstituted or substituted with at least one phosphorous, oxygen, nitrogen, sulfur or halogen atom,
n is an integer between 1 and 11,
E is selected from the group consisting of an oxygen atom, a sulfur atom, a nitrogen atom and a nitrogen atom linked to an alkyl, alkoxy or aryl group,
an outer layer consisting of units P2, which may be identical or different, corresponding to formula III:
in which:
R5 represents a hydrogen atom or one of the following groups:
alkyl, alkoxy, or aryl, unsubstituted or substituted with at least one phosphorus, oxygen, nitrogen, sulfur or halogen atom,
W represents one of the following groups:
alkyl, alkoxy, or aryl, unsubstituted or substituted with at least one phosphorus, oxygen, nitrogen, sulfur or halogen atom,
R3 and R4, which may be identical or different, represent a C1–C5 alkyl group,
X represents a hydrogen atom or a C1–C5 alkyl group or is absent, and
Z represents a halide ion, an alkylCOO− group or any other anionic group comprising carbon, oxygen, sulfur, nitrogen, phosphorus or halogen atoms, or is absent.

17 more elements

Dependencies are inferred from the claim text.

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Abstract

A polycationic phosphorus-containing dendrimer, which consists:

of a central layer in the form of a central core P0 comprising 2 to 8 functionalized groups,
of n intermediate layers, which may be identical or different, each one of said intermediate layers consisting of units P1 corresponding to formula II:
in which;
L is an oxygen, phosphorus or sulfur atom,
M represents one of the following groups:
an aromatic group di-, tri- or tetra-substituted with alkyl, alkoxy or unsaturated C1–C12 olefinic, azoic or acetylenic groups, unsubstituted or substituted with at least one phosphorous, oxygen, nitrogen, sulfur or halogen atom, or
an alkyl or alkoxy group comprising several substituents as defined when M is an aromatic group,
R1 and R2, which may be identical or different, represent a hydrogen atom or one of the following groups:
alkyl, alkoxy, or aryl, unsubstituted or substituted with at least one phosphorous, oxygen, nitrogen, sulfur or halogen atom,
n is an integer between 1 and 11,
E is selected from the group consisting of an oxygen atom, a sulfur atom, a nitrogen atom and a nitrogen atom linked to an alkyl, alkoxy or aryl group,
an outer layer consisting of units P2, which may be identical or different, corresponding to formula III:
in which:
R5 represents a hydrogen atom or one of the following groups:
alkyl, alkoxy, or aryl, unsubstituted or substituted with at least one phosphorus, oxygen, nitrogen, sulfur or halogen atom,
W represents one of the following groups:
alkyl, alkoxy, or aryl, unsubstituted or substituted with at least one phosphorus, oxygen, nitrogen, sulfur or halogen atom,
R3 and R4, which may be identical or different, represent a C1–C5 alkyl group,
X represents a hydrogen atom or a C1–C5 alkyl group or is absent, and
Z represents a halide ion, an alkylCOO− group or any other anionic group comprising carbon, oxygen, sulfur, nitrogen, phosphorus or halogen atoms, or is absent.

17 more elements