Gene Therapy Approaches

Gene therapy treats chronic diseases by introducing genes into an individual's tissues to correct missing or improper gene function.

Our Focus:

• In vivo enrichment of gene-modified cells >>>
• Bone marrow protection >>>

Gene therapy is the insertion of genes into an individual's tissues to treat disease, such as cancer where deleterious mutant alleles might be silenced, or genetic diseases wherein the faulty genes are replaced or supplemented with functional ones. The direct introduction of genes with curative effect into cells requires gene delivery vehicles, most commonly non-pathogenic derivatives of viruses, which are engineered to carry along the gene of interest into specific target cells. Depending on the type of delivery applied, the gene may be expressed only in a transient fashion, or may become integrated into chromosomal DNA. In the latter case, potentially continuous production of the therapeutic protein can be achieved, provided that the gene-modified cells yield self-renewing progeny.