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Please Note: The technology listed below is not available to the public at this time. This technology is in the early stage of research and requires further development before it is ready for the marketplace. The VA is currently in the process of identifying potential companies who may be interested in licensing and/or further developing the technology through Cooperative Research and Development Agreements (CRADA). Through cooperative research initiatives such as these, it is our hope and goal that commercial products will be fully developed and made available to benefit veterans and others.
VA TECHNOLOGY OPPORTUNITY BRIEF
Engineered Stem Cells Delivered Directly into Digestive Organs for Drug Delivery,
Gene Therapy, Cell Replacement, Research and Diagnostic Applications
(02-030)
OPPORTUNITY:
The Department of Veterans Affairs (VA) is seeking a commercial partner through a Cooperative Research & Development Agreement (CRADA) to further develop a technology that uses embryonic and adult stem cells for gene therapy, drug delivery and cell replacement in the adult digestive tract.TECHNOLOGY OVERVIEW:
In the process of embryonic development, a fertilized egg becomes a single cell with the ability to produce all of the cell types within the body. Once the cell has divided to form a blastocyst, cells in the inner mass have the potential to develop into one of three generalized tissue types-endoderm, mesoderm or ectoderm. These pluripotent stem cells are obtained from the fetal tissue of terminated pregnancies and used for establishing human pluripotent stem cell lines. They are known as embryonic stem (ES) cells.Research also has indicated the existence of multipotent cells-adult stem cells capable of generating multiple lineages of functional cells. Adult stem cells have not been found in all tissue types. However, under certain conditions in animals, these multipotent stem cells have the ability to develop into multiple types of specialized cells. Because stem cells are self-renewing and can repair damage, harnessing this ability for clinical applications has become an area of intense research.
The subject technology uses embryonic and adult stem cells for gene therapy, drug delivery and cell replacement in the adult digestive tract. These cells can be implanted into the gut and can differentiate into specialized cells such as nerves, muscles and epithelia. Before implantation, these cells can be engineered to stably express a variety of gene products. Upon implantation, they remain at the site of injection and maintain the local tissue organization. These cells also permanently express the transfected genes.
TECHNICAL MERIT:
The subject technology has several advantages:
The inventors have also genetically manipulated adult stem cells and delivered them into the gut. Such cells have integrated, although not in the same way as ES cells, and have produced NO as a gene product.
- The subject technology has several advantages:
- Permanent expression of genes manipulated in ES cells.
- Establishment of expressing ES cells within the fabric of the gut lining, avoiding the shedding of the lining that occurs every few days within the gut.
- Delivery of a previously undeliverable therapeutic nitric oxide (NO), a highly diffusible gas.
POTENTIAL APPLICATION:
The technology is targeted at diseases including inflammatory bowel disease, gastroparesis, infantile pyloric stenosis, Hirschsprung's disease and diabetes. The technology also can be used for gene therapy and cell replacement.PATENT STATUS:
A U.S. provisional patent application was filed on March 19, 2002 (60/365,155)
A US application was filed on March 19, 2003 (10/392,377).FOR MORE INFORMATION CONTACT:
Saleem Sheredos
Program Manager
Technology Transfer Program
Veterans Affairs
Office of Research & Development (12TT)
5th Floor
103 South Gay Street
Baltimore, MD 21202
202-380-5080
Fax 410.962.2141
e-mail: saleem.sheredos@va.gov