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Chapter 12: Novel Treatment Options 249 39. Walsh CE, Grompe M, Vanin E, et al. A functionally active retrovirus vector for gene therapy in Fanconi anemia group C. Blood 1994; 84: 453-459. 40. Muller LU, Milsom MD, Kim MO, Schambach A, Schuesler T, Williams DA. Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Molecular Therapy 2008; 16: 1154-1160. 41. Antonio Casado J, Callen E, Jacome A, et al. A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network. Journal of Medical Genetics 2007; 44: 241-249. 42. Jacome A, Navarro S, Casado JA, et al. A simplified approach to improve the efficiency and safety of ex vivo hematopoietic gene therapy in Fanconi anemia patients. Human Gene Therapy 2006; 17: 245-250. 43. Skinner AM, O’Neill SL, Grompe M, Kurre P. CXCR4 induction in hematopoietic progenitor cells from Fanca(-/-), -c(-/-), and -d2(-/-) mice. Experimental Hematology 2008; 36: 273-282. 44. Si Y, Ciccone S, Yang FC, et al. Continuous in vivo infusion of interferon-gamma (IFN-gamma) enhances engraftment of syngeneic wild-type cells in Fanca-/- and Fancg-/- mice. Blood 2006; 108: 4283-4287. 45. Brown BD, Gentner B, Cantore A, et al. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nature Biotechnology 2007; 25: 1457-1467.
Chapter 13 Head and Neck Squamous Cell Carcinoma in Fanconi Anemia Patients Bhuvanesh Singh, MD, PhD Introduction Head and neck cancers are among the most common tumors to develop in patients with Fanconi anemia. 1,2 Although these tumors are histopathologically similar to those in patients without FA, the frequency, distribution, and clinical course are significantly different and must be taken into account when considering cancer management in patients with FA (Table 1). This chapter provides an overview of head and neck squamous cell carcinomas (HNSCC) in the general population in contrast to those occurring in FA patients, with a focus on aspects of prevention, treatment, and surveillance in FA patients. Head and Neck Cancer in the General Population Head and neck cancer is a group of diseases linked together by a common histopathology, squamous cell carcinoma (SCC). These diseases can occur anywhere in the mucosal linings of the upper aerodigestive tract, beginning in the oral cavity and nasopharynx, and extending to the oropharynx, larynx or hypopharynx. About 30,000 new patients present annually with head and neck cancer in the United States and about 30% succumb to their disease. Internationally, head and neck
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Chapter 4 Gastrointestinal, Hepatic
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Chapter 7 Endocrine Disorders in Fa
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