Effective vector platform for gene transfer and gene therapy

摘要

The invention relates to the discovery that mutations of serine residues of an AAV capsid results in significantly greater transfection efficiency than the wild type AAV2 virus. In one embodiment, the present invention provides a method of improving efficiency of gene transfer and/or gene therapy to a cell by inhibiting phosphorylation of one or more serine residues of a virus capsid protein, where the inhibition of the phosphorylation of one or more serine residues results in a decrease of ubiquitination of the virus capsid protein in the cell. In another embodiment, one of the one or more serine residues is Serine 264. In another embodiment, the Serine 264 residue is mutated to Alanine (S 264 A).

主权项

1. A vector, comprising:
an isolated adeno-associated virus (AAV) capsid comprising SEQ. ID. NO.: 2 or SEQ ID NO: 3 that resist ubiquitination.