About This Project

We are engineering a bacteriophage system that naturally assembles DNA ministrings - a safe & effective gene therapy vector to encode any gene(s) for any therapeutic targeted application. Bacteriophage Expression and Assembled Ministrings (BEAM) vectors combine the proven superior safety and efficacy of DNA ministrings with the efficiency and simplicity of phage production. If successful, it is poised to become the new gold standard vector for gene therapy design and production.

Ask the Scientists

What is the context of this research?

Gene therapy is the treatment of disease using sequences of DNA or RNA (genes). To get a gene into a cell where it can exert a therapeutic effect, it needs to be packaged into a delivery vector. Most current vectors are modified human viruses, which are efficient, but can be unsafe. While safer, non-viral methods tend to be much less efficient. Our DNA ministring technology has been scientifically proven to offer the safest, most efficacious and most efficiently scale-able non-viral gene therapy vector system.

What is the significance of this project?

Non-viral gene therapy strategies are mostly quite ineffective, until DNA ministrings, but generally prove safer than viral systems using engineered adenoviral or lentiviral vectors such as HIV. In contrast, viral systems are effective, but pose dangers such as immunogenicity and mutagenesis.

Our goal with this project is to produce a one-step scalable production of a target-able genetic therapeutic (BEAM). Phages can be easily modified for different delivery options, are physically and thermally stable, and can be produced in large quantities at greatly reduced cost compared to other vectors. BEAM also offers simple purification that lowers production costs. Stable and cheap? It's perfect for the design and delivery of new therapies to developing countries, our mission.

What are the goals of the project?

We are constructing BEAM as a proof of principle to demonstrate the capabilities of phage as gene therapy vectors.

Our goals are simple:

1) Develop the parent plasmid that will generate DNA ministrings in the bacterial host of the bacteriophage. This plasmid will carry the gene of interest, factors to direct gene expression in human cells, and sites to direct phage assembly in the bacterial host.

2) Construct the second plasmid platform on which ministring and phage will self-assemble into BEAM particles inside the cell with no additional effort, the sophistication of biological simplicity...and that's our motto.

3) Test the system and ensure that BEAM particles are being produced and work.