Laget, Inc. - Light Activated Gene Therapy

The LAGeT Laser
The promise of gene therapy is the ability to efficiently transfer new genetic material into patient cells to either replace defective genes or introduce therapeutic genes in order to treat disease. Much progress has been made in the discovery of therapeutic genes that prevent, cure or arrest disease. But the success of gene therapy is limited by our ability to deliver therapeutic genes in a site-specific, safe, and effective manner. In other words, if the therapeutic gene represents a magic bullet, we still need the magic gun to fire the magic bullet. It’s in this area that LAGeT has developed its breakthrough technology.
	Viral vectors provide one of the most promising approaches for efficient gene therapy delivery. However, side effects and efficacy issues have plagued a number of the viral vectors currently being used. One viral vector has moved to the forefront as the gene therapy vector of choice because of its favorable safety profile. Adeno-Associated Virus, or AAV, offers a number of advantages over alternative vectors, including: the ability to deliver genes into a variety of cell types, a favorable profile for manufacture, storage and handling, and the ability to administer AAV in vivo because of the absence of tumorogenic or immune responses attributable to the vector.
It is for these reasons that LAGeT is concentrating its efforts on AAV, through the design of an ultraviolet (UV) laser, which greatly enhances the utility of AAV as a gene therapy vector. The LAGT laser overcomes a major challenge facing AAV gene therapy by initiating a cellular response, which leads to the expression of the therapeutic protein encoded by the vector. Such control over the expression of the therapeutic gene is achievable because rAAV is a single stranded DNA virus. The single DNA strand is not functional, containing only half of the blueprint necessary for the expression of the therapeutic protein. For this blueprint to be completed, a second strand of DNA must be synthesized to form active, double-stranded DNA. In most circumstances, the single-stranded DNA will not spontaneously form double-stranded DNA. However, pre-treatment of the target area with the LAGT laser alters the cellular environment to allow for efficient formation of the second DNA strand.

LAGeT is focused on developing gene therapeutic approaches for musculoskeletal diseases, including articular cartilage defects a leading cause of osteoarthritis. LAGeT hopes to be able to develop treatments for the many individuals who now suffer from these painful and often debilitating diseases, dramatically improving their quality of life.