title: Induced Pluripotency and Gene Editing in Fanconi Anemia.
abstract: Induced pluripotent stem cells (iPSCs) represent an invaluable tool in a chromosomal instability syndrome such as Fanconi anemia (FA), as they can allow to study of the molecular defects underlying this disease. Many other applications, such as its use as a platform to test different methods or compounds, could also be of interest. But the greatest impact of iPSCs may be in bone marrow failure diseases, as iPSCs could represent an unlimited source of autologous cells to apply in advanced treatments such as gene therapy. At the same time, genome editing constitutes the next generation of technology to further develop a safer, personalized, targeted gene therapy. Despite the promising advantages that these two technologies would present in a disease such as FA, the specific characteristics of the disease make both of these processes especially challenging. Efficient and safer FA-hiPSC (human induced pluripotent stem cell) generation methods, robust and reliable differentiation protocols for iPSCs, as well as really efficient delivery methods to perform targeted gene correction should be developed.
authors.forename: Susana
authors.initials: S
authors.lastname: Navarro
authors.forename: Alessandra
authors.initials: A
authors.lastname: Giorgetti
authors.forename: Angel
authors.initials: A
authors.lastname: Raya
authors.affiliation: Pediatric BMT, MMC 366 Mayo, 420 Delaware St SE, Minneapolis, MN 55455, United States.
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
mesh_terms: D000818:Animals; D002454:Cell Differentiation / Q000235:genetics; D005199:Fanconi Anemia / Q000235:genetics* / Q000628:therapy*; D005347:Fibroblasts / Q000166:cytology / Q000378:metabolism; D000072669:Gene Editing / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000378:metabolism
keywords: Fanconi anemia; Gene editing; Gene therapy; Induced pluripotent stem cells