Yanina Rozenberg - Solano Beach CA, US Viacheslav Medvedkin - Los Angeles CA, US Natalia Fedorovna Medvedkina - Pushchino, RU Alexander Viacheslavovich Medvedkin - Pushchino, RU W. French Anderson - San Marino CA, US
International Classification:
A61K 31/70 A61P 43/00 C12N 15/00
US Classification:
514 44, 4353201
Abstract:
Novel and improved compositions and methods for gene therapy are provided. In particular, a targeted artificial gene delivery (“TAGD”) vehicle is provided, comprising a multifunctional artificial surface moiety surrounding a recombinant viral particle (nucleocapsid) or recombinant core for gene delivery.
The invention provides improved vectors for cell-specific gene delivery to a target cell. The vectors according to the instant invention comprise a recombinant core containing the genetic materials to be delivered and an artificially reconstituted surface encompassing the core. The surface facilitates targeting and cell fusion of the vector, and also provides an immunoprotection function for the vector. Methods for preparing the sectors and for transfecting eukaryotic cells using the vectors also are disclosed. Gene therapy has received, great deal of attention for its potential of providing effective treatment of many human diseases, ranging from rare heritable genetic defects and common diseases such as cancer, AIDS, hypertension, atheroma and diabetes. The great potential of gene therapy has up until now been hampered by the lack of efficient vector systems for delivery of genetic constructs into cells in vivo and ex vivo. As with the targeted delivery of conventional drugs, one of the goals of genetic therapy is to maximize the local therapeutic effect of gene delivery while minimizing the potenial for systemic adverse events. To achieve this goal, an ideal gene delivery vector should possess several attributes. First, the vector should be able to reach a target site within/the organism, and preferably should be able to recognize the specific cell types. This requires that the vector have low immunogenicity as well as targeting results demonstrate that re-enveloping of the core with artificial surfaces that contain fusion and retargeting molecules can be achieved.