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Chapter 11: Late Effects in FA Patients Post-Transplant 235 11. Rosenberg PS, Socie G, Alter BP, Gluckman E. Risk of head and neck squamous cell cancer and death in patients with Fanconi anemia who did and did not receive transplants. Blood 2005; 105: 67-73. 12. Wagner JE, Tolar J, Levran O, et al. Germline mutations in BRCA2: shared genetic susceptibility to breast cancer, early onset leukemia, and Fanconi anemia. Blood 2004; 103: 3226-3229. 13. Guardiola P, Socie G, Li X, et al. Acute graft-versus-host disease in patients with Fanconi anemia or acquired aplastic anemia undergoing bone marrow transplantation from HLA-identical sibling donors: risk factors and influence on outcome. Blood 2004; 103: 73-77. 14. Masserot C, Peffault de Latour R, Rocha V, et al. Head and neck squamous cell carcinoma in 13 patients with Fanconi anemia after hematopoietic stem cell transplantation. Cancer 2008; 113: 3315-3322.
Chapter 12 Novel Treatment Options Jakub Tolar, MD, PhD Opportunities and Challenges Physicians have made remarkable progress in hematopoietic cell transplantation over the last decade, and the care of FA patients in need of transplantation has improved dramatically. A minority of patients, however, cannot be transplanted because of their medical conditions or do poorly during or after transplant. Therefore, we aim to investigate all possibilities that can translate into better care for FA patients. The prime candidates in this realm are gene therapy, stem cell therapy, and stem cell gene therapy. Gene Therapy Gene therapy vectors Delivering a gene into a cell is not a trivial matter. Investigators in the past have realized this and have used viruses as vectors for this purpose. Viruses have developed their own means of delivering their genes into cells, and researchers have “borrowed” these properties to insert genes of interest into cellular genomes. The retroviral vector is the traditional vector, although improved lentiviral vectors, with an added advantage of being able to transduce non-dividing cells, have been used recently. Small DNA viruses, called pyroviruses, represented by adenovirus and adeno-associated virus, represent another group of viruses that have been investigated in great detail in preclinical gene therapy testing.
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Chapter 4 Gastrointestinal, Hepatic
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Chapter 7 Endocrine Disorders in Fa
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Chapter 9 Matched Sibling Donor Hem
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