Cell And Gene Therapy
Center Leader: Katherine High, MD, Howard Hughes Medical Institute Investigator
Cell therapy, gene therapy and stem cell research offer unprecedented opportunities for developing new medical therapies for serious diseases that affect children. Current research in these areas may contribute to a new approach to treating disease – regenerative medicine – where defective genes, cells or even entire organs are replaced.
One of the most exciting frontiers in modern medicine is the concept of using a gene to treat a genetic disease. Researchers have made dramatic progress in innovative gene therapy strategies that could cure or even prevent disease. The idea of introducing corrective genes into cells to stop a disease at its source – such as hemophilia, cystic fibrosis and congenital blindness – is rapidly becoming a reality.
Cell therapy is an older discipline that has been modified to include blood transfusions, organ and bone marrow transplantation and modern adoptive transfer of white blood cells to treat cancer. Recent developments have been focused on using stem cells to replace defective tissues and cure disease. While this work is in the earlier stages of research, the possibility of taking a person’s own cells and using them as stem cells to treat that person’s disease is an exciting frontier.
A sizable group of individuals at Children’s Hospital are focused on these areas of investigation and are members of the Cell and Gene Therapy Research Affinity Group, which aims to develop new treatment modalities for children with inherited and infectious diseases.
The affinity group fosters a multidisciplinary approach among investigators working to discover new gene and cell therapies in search of cures for debilitating and life threatening childhood disorders. This approach may lead not only to a variety of applications in the treatment of inherited disorders such as hemophilia, muscular dystrophy and cystic fibrosis, but also to the treatment of acquired and complex disorders, including diabetes, heart disease, infectious diseases, neurodegenerative disorders and cancer.
One of the most notable activities of the affinity group is the extremely high-quality seminar series that attracts distinguished outside speakers who are active in the cell, gene and stem cell research field. Speakers not only hold public seminars, but also visit with individual lab members to discuss specific research projects. In addition to affinity group seminars, the group actively pursues collaborative funding.
Cell and gene therapy affinity group members have successfully competed for NIH Program Project grants, including an award entitled Gene Therapy for Hemophilia, which investigates the use of vectors introduced into either skeletal muscle or circulating blood platelets as a means of treating hemophilia. Another program project is Immune Responses in Gene Therapy for Hemophilia which focuses on the body’s immune responses in the setting of gene therapy for hemophilia.