About This Project
Most of the approaches to treating HIV are pharmacological in nature. But modern medicine has yet to identify a medication that will knock out HIV entirely. An alternative approach is to deliver genes to protect cells from HIV infection. However, scientists have yet to develop a safe, effective, and long-lasting way to deliver the therapeutic genes into a living patient with only one or two treatments.Ask the Scientists
Join The DiscussionWhat is the context of this research?
Numerous genetic approaches to fighting HIV have
been tested and found to work in the laboratory. The techniques for introducing genetic material into a cell in a Petri dish have been around for decades. As yet, however, there is no way of safely delivering these treatments on a long-term basis into a living human. VG's vision is a delivery system that will safely confer lasting protection against the HIV virus with a single dose, for uninfected and infected persons alike. The delivery system makes use of a complicated regulatory system to turn on the defensive genes when HIV is present; and then turns itself off when the HIV is gone. While the infection is present, the delivery system is able to reach new T-cells to equip them with the defensive genes.
What is the significance of this project?
Anti-retroviral treatments (ARVs) cost approximately $15,000 per person per year (pppy) in North America; $3000-5000 in India and Africa. These medications have to be administered indefinitely, since they have yet to successfully eliminate HIV from every tissue reservoir that it hides in.
A genetic therapy which works after a single administration will free up hundreds of billions of dollars which can then be re-invested into communities that have been devastated by HIV; also into treatments for other diseases that have been eclipsed by the HIV pandemic. And furthermore, our vector is a "platform technology," meaning that its delivery mechanism might be re-deployed to target other viruses.
What are the goals of the project?
In order to realistically qualify for NIH funding, a grant application has to include what's called "prior data." - i.e. preliminary experimentation to prove that some critical aspect of the project will work.
ImQuest has agreed to perform this research, provided VectorGen supplies the genetic material for testing. So the immediate goals for this project are listed below:
1) We will have a genetic vector with the appropriate regulatory elements engineered.
2) We will have the gene coding for the therapeutic molecule manufactured, and inserted into the vector.
3) We will demonstrate that the regulatory elements encoded in the vector will function as intended. That is:
a) The therapeutic gene will turn on when HIV is present
b) The vector will shut itself down when HIV is gone
Budget
VG has partnered with ImQuest, a major biotechnology firm. ImQuest has agreed to perform critical experimentation to validate the design of our vector. According to the agreement VG will be allowed to retain the Intellectual Property rights if we purchase the genetic material that we are planning to test. These days, it is possible to simply synthesize genes that code for a molecule with specific properties. These genes can be purchased from gene manufacturing companies. We'll also need some genetic material (called plasmids) to show that our therapeutic molecule will perform its function as expected.
Some of the money we raise will go to ImQuest to compensate them for laboratory materials (cell growth media, tissue culture dishes, etc). We are also requesting money to cover the cost of applying for a provisional patent on our delivery system.
If you are interested in learning more about our technology, please feel free to visit us at our website: www.vectorgen.com.
Meet the Team
Team Bio
Laura Prendergast is fundamentally a Cell and MolecularBiologist. She received her BA in Biology from Columbia University (where she was trained by the likes of Nobelist Marty Chalfie), and an MS in Recombinant DNA Technology from New York University. Besides Gene Therapy, she is extremely well-versed in a number of sub-disciplines in the field of Biology, including Neurobiology. The original idea for the genetic vector we are proposing to develop occurred while studying the lambda repressor in Molecular Biology of the Gene, the genetics textbook by James Watson.
Laura Prendergast
Laura Prendergast is fundamentally a Cell and Molecular
Biologist. She received her BA in Biology from Columbia University (where she was trained by the likes of Nobelist Marty Chalfie), and an MS in Recombinant DNA Technology from New York University. Besides Gene Therapy, she is extremely well-versed in a number of sub-disciplines in the field of Biology, including Neurobiology. The original idea for the genetic vector we are proposing to develop occurred while studying the lambda repressor in Molecular Biology of the Gene, the genetics textbook by James Watson.
Lab Notes
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Additional Information
Project Backers
- 7Backers
- 22%Funded
- $535Total Donations
- $76.43Average Donation