SC & Gene Therapy

The success of whole marrow transplantation for the correction of several genetic disorders has focused attention on the hematopoietic stem cell as a target for gene therapy for the correction of several genetic disorders of hematopoiesis and metabolism. Precisely how to manipulate the stem cell to achieve efficient gene transfer without compromising the functional potential required for long-term hematopoiesis remains a critical area of research. Studies have demonstrated that retroviral-mediated gene transfer into HSCs is feasible and safe. However, currently available HSC gene-transfer protocols do not reliably transfer genes into HSCs with long-term repopulating capacity. A greater understanding of the basic biology of retroviruses and hematopoiesis will enhance the development of more advanced and efficient HSC retroviral vector delivery systems. Successful HSC gene transfer approaches will need to develop methods of targeting nondividing HSCs and/or stimulating HSCs to proliferate prior to transduction. New generation of vectors may permit the inclusion of larger genes, transcriptional regulatory units, tissue specific expression, and multiple genes and allow the insertion of viral genes into site-specific genomic locations.

The following review  Stem Cell Directed Gene Therapy is sure to tell you a lot about the relationship between these two hotspots!

For more, you can go to the column Gene Therapy  or use the navigation system in CMBI.