The present invention provides a new therapeutically very efficient vector for gene therapy that allows the reduction of viral load and/or number of infections needed in order to achieve the same effectiveness of the same vector carrying the therapeutic protein only.
| Patent title | Innovative strategy to enhance the efficiency of brain gene therapy |
|---|---|
| Thematic area | Health |
| Ownership | Oregon Health & Science University, ALMA MATER STUDIORUM - UNIVERSITA' DI BOLOGNA |
| Inventors | Claudia Fuchs, Stefania Trazzi, HOROYUKI NAKAI, Elisabetta Ciani |
| Protection | Italy, China, Europe, USA |
| Licensing status | Available for development agreements, option, license and other exploitation agreements |
| Keywords | Biodistribution, Therapeutic protein, Drug delivery, Gene therapy |
| Filed on | 13 June 2019 |
Gene therapy can be broadly defined as the “transfer of genetic material” to cure, to prevent or to ameliorate a disease. One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells. Gene therapy typically involves the insertion of a functioning gene into cells to correct a cellular dysfunction or to provide a new cellular function. Until a decade ago, strategies for gene delivery to the brain were limited mostly to stereotaxic injection of viral vectors to the brain, and widespread gene delivery was achieved through the use of multiple injections. But recently, there is a strong need to ameliorate the effectiveness of gene therapy while not increasing or even decreasing the strongly adverse side effects that are potentially triggered by the use of gene therapy vectors that are integrated in the host genome.
The invention relates to a modified vector for gene therapy composed of different nucleic acid encodings, which allows to reduce the viral load and / or the number of infections necessary to obtain the same efficacy when compared to the same vector carrying only the therapeutic protein. The most prominent central nervous system (CNS) gene delivery vector is currently the adeno-associated virus (AAV). The modified gene therapy vector of the invention provides an increased therapeutic activity compared to the same vector before the modification because the produced therapeutic protein is vehiculated outside the genetically modified cell, and is internalized in cells that are not genetically modified, thereby providing a therapeutic effect also in cells that have not been modified by the vector, hence amplifying, the therapeutic effect with respect to the number of cells infected.
Applications:
- brain gene therapy;
- targeted drug delivery
Advantages:
- reduction of viral load;
- reduction of number of infection.