Towards gene therapy of sickle cell disease

Abstract

Sickle cell disease (SCD) is a significant health problem in the United States, in part of Europe and in many developing countries. Progress has been made in the management of the disease but a cure is still elusive except in the rare cases where bone marrow transplant is a practical option. Hopes for a complete universal cure rest mostly on two gene therapy approaches: correcting the mutation that causes the disease, or adding a corrective gene to the genome of the patient. The former approach is extremely attractive but is in its early stages of development. One of the most important remaining obstacles for the latter approach is the control of expression of a transgene integrated at ectopic chromosomal sites. Animal models for SCD should help in the development of the gene therapy methods discussed above, as well as the pharmacological approaches that aim at ameliorating or eliminating the symptoms of SCD.

• anti-sickling globins
• cre recombinase
• gene therapy
• insulator
• recombinase-mediated cassette exchange
• sickle cell disease