Inherited Genetic Diseases Treatable With Stem Cells

Our long experience with prenatal diagnosis and planning for optimum care allows us to offer the highest quality treatment whether intervention is indicated before or after birth.

Under the direction of Dr. Michael Harrison, the Fetal Treatment Center was the first institution to develop fetal surgery techniques. The first open fetal surgery in the world was performed at UCSF since the early 1980's. We presently have more experience with fetal surgery and endoscopic fetal intervention (FETENDO fetal surgery) than any other institution in the world. We are dedicated to stem cell research and treatment innovation.

What genetic diseases are potentially treatable with stem cells?

There are a large number of inherited genetic diseases (like sickle cell anemia) that can now be accurately diagnosed early in fetal life by examining fetal DNA obtained by chorionic villus sampling or amniocentesis. In most cases the testing is done because the disease has been identified to run in the family.

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What is the outcome for a fetus with an inherited genetic disease?

Because stem cell defects result in such a wide of variety of diseases (hemoglobinopathies, immunodeficiencies, inborn errors of metabolism, etc.), the outcome for the fetus depends on that particular disease. The wide range of  outcomes are anywhere from death or severe life-long impairment to survival and treatment after birth with minimal impairment. 

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How serious is the inherited genetic disease in my fetus?

For you to make the best decision, you will need accurate and complete information about your fetus’s condition, including the type and severity of the defect determined by DNA testing. In addition, you will want to know if there are associated defects, or if this problem is part of a cluster of problems (syndrome). 

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What are my choices during this pregnancy?

The most important consideration is the accuracy of the diagnosis through genetic counseling about the consequences of that particular inherited stem cell defect. If after full and complete counseling about the defect and its consequences, you choose to continue the pregnancy, you will start planning the proper time and place of delivery for optimal care after birth. For inherited stem cell defects that cause trouble at birth and require immediate treatment, the place of delivery will be the most important consideration.

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What will happen after birth?

Management after birth will depend on the nature of the inherited genetic defect. In many cases, very specialized therapy by experts in that disease is only available at highly specialized tertiary care centers. For many diseases, hematopoietic stem cell transplantation, usually in the form of a bone marrow transplant, will be planned after birth. Often, repeated testing and careful follow-up are necessary to protect the baby until the transplant is performed.

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