Methods of preparing bone marrow stromal cells for use in gene therapy

Patent Number: 6991787

This patent involves using gene-transfected bone marrow cells as a way of delivering the gene of interest to a tissue that needs it. For example, lets take stroke patients. Conceptually one could administer a therapeutic gene directly into the area of cell death, or in proximity to it, in order to prevent further cell death or to induce regeneration of neurons. This approach is complex since one would need to open up the cranium, or somehow insert the gene in the brain, which is relatively invasive. The fact that after stroke, or heart attack, or other types of tissue damage, there is a migration of bone marrow derived cells into the area of damage. Accordingly, if one takes cells of a similar phenotype, transfects them with a gene, and introduces them into the patient with injured tissue, it would be reasonable to believe that the stem cells will migrate to where they are needed, and release the product of the gene that they are transfected with. The current patent teaches a method of storing cells that have been transfected for this use. The patent has 1 independent claim covering a method of storing cells that have been transfected. One example of using bone marrow cells as gene vectors is discussed in a publication by the authors of the patent (Hurwitz DR et al. Systemic delivery of human growth hormone or human factor IX in dogs by reintroduced genetically modified autologous bone marrow stromal cells. Hum Gene Ther. 1997 Jan 20;8(2):137-56.)

View this patent on the USPTO website.