Recently several systems including viral and non-viral carriers that can be used to transfer foreign genetic material into cells have been developed with the aim of enhancing gene transfer in vivo. Non-viral vectors are relatively easy to produce in clinically relevant quantities, and associated with fewer safety concerns. Furthermore, synthetic non-viral vectors provide flexibility in formulat...

We introduced replicase genes of brome mosaic virus (BMV) to Nicotiana tabacum cv. Petit Habana (SR1) using two different types of transformation vectors containing cDNAs of BMV RNA 1 and RNA 2. One type (V type) contains cDNA from which complete viral RNAs are transcribed. These RNAs can function as templates for viral replicase. The other type (M type) contains cDNA from which viral RNAs with...

Safety in gene therapy is an important issue since both viral and non-viral vectors have toxic side effects. Not only vectors themselves, but also distributions of produced proteins affect safety in gene therapy; thus, development of target-selective gene transfer methods is rational. We have developed organ-, region- and cell-selective gene transfer methods using non-viral vectors. To deliver ...

The ability to mediate targeted and specific delivery of therapeutics to cancer cells remains one of the most important hurdles in effectively treating cancer. This aspect also remains as one of the greatest limitations of gene therapy as well. Targeted vectors based on the use of DNA-binding agents attached to cell specific ligands or "molecular conjugates" were created with the goal of over-c...

PURPOSE Non-viral vectors have been widely proposed as safer alternatives to viral vectors, and cationic polymers have gained increasing attention because they can form self-assembly with DNA. Chitosan is also considered to be a good candidate for gene delivery systems, since it is already known as a biocompatible, biodegradable, and low toxic material with high cationic potential. However, low...

Over the last few decades, as a promising strategy for the treatment of many refractory diseases, such as inherited diseases (Martin-Rendon & Blake, 2003) and acquired immunodeficiency syndrome (AIDS) (Fanning et al., 2003), gene therapy, the objective to allow a gene to express the protein coded in the target cells and consequently to treat disease by the protein secreted from cells transfecte...

Along with viral vectors, non-viral strategies have been developed in order to efficiently deliver nucleic acids to ocular cells. During the last decade, we have observed that the outcome of these non-viral delivery systems depends on the genetic material used, the targeted tissue or cells, the expected effect duration, and the routes of administration. Assessment of efficiency has been evaluat...

The unique diversity of parvoviral vectors with innate antioncogenic properties, autonomous replication, ease recombinant vector production and stable transgene expression in target cells makes them an attractive choice as viral for gene therapy protocols. Amongst various parvoviruses that have been identified so far, originating from adeno-associated virus, minute virus mice (MVM), LuIII parvo...

In vivo gene transfer of recombinant E1-deficient adenoviruses results in early and late viral gene expression that elicits a host immune response, limiting the duration of transgene expression and the use of adenoviruses for gene therapy. The prokaryotic Cre-lox P recombination system was adapted to generate recombinant adenoviruses with extended deletions in the viral genome (referred to here...

Progression of RNA interference-based gene silencing technologies for the treatment of disorders of the central nervous system (CNS) depends on the availability of efficient non-toxic nanocarriers. Despite advances in the field of nanotechnology undesired and non-specific interactions with different brain-cell types occur and are poorly investigated. To this end, we studied the cytotoxic and ne...