Chaired by Dennis D. Hickstein, NCI

Conference Room F1/F2, Natcher Conference Center

Correction of monogenetic diseases using gene therapy continues to beguile investigators and the public, offering the apparent simplicity of gene replacement with the hidden perils of lethal adverse events ranging from the host response to foreign vectors to the risk of insertional mutagenesis leading to leukemia.  Nowhere is this dichotomy more apparent than in genetic immunodeficiency diseases where the field has seen its greatest successes and its most dramatic set-backs.  In this session the target immunodeficiency diseases-chronic granulomatous disease or CGD, leukocyte adhesion deficiency or LAD, and adenosine demainase deficiency or ADA-SCID will be described, along with the gene therapy approaches aimed at treating these diseases currently underway in the Clinical Research Center on the NIH campus.  The now well-recognized risks of insertional mutagenesis with the current vectors for gene delivery will be described as well.

Program:

Safety/Efficacy Profiles of Hematopoietic Stem Cell Therapies for Chronic Granulomatous Disease: Transplant or Gene Therapy
Harry Malech, NIAID

Gene Therapy Approaches for Leukocyte Adhesion Deficiency Using the Canine Leukocyte Adhesion Deficiency Model
Dennis D. Hickstein, NCI

Gene Therapy for Children with Adenosine Deaminase Deficiency SCID
Fabio Candotti, NHGRI

Insertional Mutagenesis and Hematopoietic Stem Cell Gene Therapy
Cindy Dunbar, NHLBI