Gene Therapy for Fanconi Anemia

Exclusion Criteria

• •Non-hematopoietic malignancy where the expected survival is less than 2 years
• •Myelodysplastic syndrome as defined by World Health Organization (WHO) criteria
• •Acute myeloid leukemia as defined by WHO criteria
• •Pregnancy or lactation; females of childbearing potential and males who are admitted to the study will be advised that the study procedures and study drugs may be teratogenic, and they will be required to take adequate measures to prevent conception for the duration of the study
• •Concurrent enrollment in any other study using an investigational drug
• •Physical or emotional status that would prevent informed consent, protocol compliance, or adequate follow-up
• •Patients for whom an human leukocyte antigen (HLA) matched sibling donor bone marrow transplant is being actively pursued will not be eligible for study until it is determined that no sibling donor is available or that a stem cell transplant is not feasible during the time the patient might be on study
• •* No patient will be included in this study as an alternative to a clinically indicated HLA matched sibling donor stem cell transplant
• •* If an HLA matched sibling donor is identified, but stem cell or marrow collection is not feasible (e.g., donor is in utero, is a newborn from whom cord blood was not collected, or is unable to undergo a donation procedure because of ill health), a patient may be included in the study at the discretion of the investigators
• •Significant associated diseases including documented human immunodeficiency virus (HIV) infection, uncontrolled hypertension (diastolic blood pressures \> 95%ile for age), unstable angina, congestive heart failure (\> New York \[NY\] class II), poorly controlled diabetes (hemoglobin A1c \[Hgb A1c\] \> 7%), coronary angioplasty within 6 months, myocardial infarction within the last 6 months, or uncontrolled atrial or ventricular cardiac arrhythmia, abnormal coagulation, persistent abnormal urinalysis reflecting intrinsic renal disease
• •Active ongoing viral, bacterial, or fungal infection