| Grant number: | 12/05066-7 |
| Support Opportunities: | Scholarships in Brazil - Master |
| Start date: | July 01, 2012 |
| End date: | June 30, 2014 |
| Field of knowledge: | Health Sciences - Medicine |
| Principal Investigator: | Bryan Eric Strauss |
| Grantee: | Marlous Vinícius Gomes Lana |
| Host Institution: | Instituto do Câncer do Estado de São Paulo Octavio Frias de Oliveira (ICESP). Coordenadoria de Serviços de Saúde (CSS). Secretaria da Saúde (São Paulo - Estado). São Paulo , SP, Brazil |
Abstract The recombinant adeno-associated virus (rAAV) have several advantages which contribute to their increasing use in gene therapy. Broad tropism, ability to transduce cells post-mitotic or terminally differentiated, low frequency integration, immune response moderate and high titer sustained expression is rAAV characteristics that contribute to the prospect of its application safe and successful in a variety of strategies for gene therapy. Production of rAAV particles may be accomplished by several protocols that are typically very difficult to perform on a large scale. This is due to the complexity of the biology of AAV and the adjustments that are incorporated into rAAV vectors. For example, the use of a helper virus such as adenovirus, provides factors essential for replication of the rAAV. However, contamination of the rAAV stock with adenovirus is not desired and may hinder the use of rAAV. In another example, the use of helper adenovirus can be replaced by use of a plasmid encoding only the minimal adenoviral factors required to support replication of the rAAV. In this case, the production of rAAV involves co-transfecting three plasmids (which encodes a gene of interest, the other providing rep and cap genes of AAV and the third providing helper functions). The problem with this approach is the procedure of transition from plasmids, a protocol that is difficult to perform large scale large scale. The ultimate goal of this work is to develop a production method of Adeno-associated viral vector independent of the transfection of plasmids and to minimize the risk of contaminating the rAAV stocks with helper viruses. This current proposal is described assays a proof of principle that the use of RNAi against adenoviral genes hexon and IVa2 eliminate contamination of the rAAV stock with the adenovirus helper. The success of this first step is critical to further build an improved system for producing rAAV which also does not involve transfecting plasmids. | |
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