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Gene Therapy continuous to offer hope for novel treatment of a variety of acquired and inherited diseases. However, much research, development and trials are needed to translate the basic principles of gene transfer into practical and safe treatment regimens.
INHERINET aims to research and develop gene therapy for selected inherited diseases and to expand the success that has been achieved for one disease, X-linked severe combined immune deficiency.
The diseases selected involve those that, in principle, can also be therapeutically approached by blood stem cell transplantation. These stem cells, which reside in the bone marrow, are uniquely capable of generating an entirely new blood cell production and immune system when transplanted. Blood stem cells are close to ideal targets for gene therapy:
Inherited diseases that affect blood cell production and the immune system together are among the most common inherited diseases of humans
Blood stem cells can be easily obtained and transplanted,
Blood cells come everywhere in the body, thereby delivering their products, which means that also diseases that do not directly affect blood cells may be approached by blood stem cell gene therapy
Bone marrow stem cells are rare and occur in a frequency of 1 per 100,000. Although purification of stem cells in experimental settings has been possible, in practice they are not enriched further than approximately 100-fold. This means that the vehicle for gene transfer should be highly efficient and preferably selective for stem cells. Efficient vehicles for gene transfer are certain classes of viruses, the retroviruses, which are capable of delivering healthy genes to the genome of diseased cells. INHERINET’s researchers are focused on increasing the efficiency and improving the safety of these vehicles, which is a prerequisite for clinical gene therapy.
To develop gene therapy, other hurdles are addressed as well, such as:
The quality of the repaired blood stem cells, in other words, are these cells still capable to provide a life long active blood cell and immune system
The possible immune reactions against the transgene product
Engraftment and production of gene modified cells in case the transgene product does not confer a natural selective advantage
Recommended further reading:
Human Genome Project Information – Gene Therapy
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