Gene Therapy for the Treatment of Fanconi's Anemia Type C
Status:
Completed
Trial end date:
2009-02-11
Target enrollment:
Participant gender:
Summary
Fanconi's Anemia is an inherited disorder that can produce bone marrow failure. In addition,
some patients with Fanconi's anemia have physical defects usually involving the skeleton and
kidneys. The major problem for most patients is aplastic anemia, the blood counts for red
blood cells, white blood cells, and platelets are low because the bone marrow fails to
produce these cells. Some patients with Fanconi's anemia can develop leukemia or cancers of
other organs.
Many laboratory studies have suggested that Fanconi's anemia is caused by an inherited defect
in the ability of cells to repair DNA. Recently, the gene for one of the four types of
Fanconi's anemia, type C, has been identified. It is known that this gene is defective in
patients with Fanconi's anemia type C.
Researchers have conducted laboratory studies that suggest Fanconi's anemia type C may be
treatable with gene therapy. Gene therapy works by placing a normal gene into the cells of
patients with abnormal genes responsible for Fanconi's anemia type C. After the normal gene
is in place, new normal cells can develop and grow. Drugs can be given to these patients kill
the remaining abnormal cells. The new cells containing normal genes and will not be harmed by
these drugs.
The purpose of this study is to test whether researchers can safely place the normal
Fanconi's anemia type C gene into cells of patients with the disease. The gene will be placed
into special cells in the bone marrow called stem cells. These stem cells are responsible for
producing new red blood cells, white blood cells, and platelets.