Method of targeted gene delivery using viral vectors

Details Method of targeted gene delivery using viral vectors Method of targeted gene delivery using viral vectors

C - Chemistry, Metallurgy

12

N

C12N 7/01 (2006.01) C12N 15/867 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2611056

Methods and compositions are provided for delivering a polynucleotide encoding a gene of interest to a target cell using a virus. The virus envelope comprises a cell-specific binding determinant that recognizes and binds to a component on the target cell surface, leading to endocytosis of the virus. A separate fusogenic molecule is also present on the envelope and facilitates delivery of the polynucleotide across the membrane and into the cytosol of the target cell. The methods and related compositions can be used for treating patients having suffering from a wide range of conditions, including infection, such as HIV; cancers, such as non-Hodgkin's lymphoma and breast cancer; and hematological disorders, such as severe combined immunodeficiency.

L'invention concerne des procédés et des compositions permettant de transférer un polynucléotide codant pour un gène particulier dans une cellule cible au moyen d'un virus. L'enveloppe virale comporte un déterminant de liaison spécifique de la cellule, qui reconnaît un composant de la surface de la cellule cible, et se lie à celui-ci, ce qui conduit à l'endocytose du virus. Une molécule fusogène distincte est en outre présente sur l'enveloppe et facilite le transport du polynucléotide à travers la membrane et dans le cytosol de la cellule cible. Ces procédés, et les compositions correspondantes, peuvent être utilisés pour le traitement de patients présentant un large éventail de pathologies, notamment des infections, telles que l'infection VIH, des cancers tels que le lymphome non-hodgkinien ou le cancer du sein, et des affections hématologiques, telles que l'immunodéficience combinée sévère.

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