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.
Bibliographical noteFunding Information:
Work conducted at the Hematopoietic Innovative Therapies at the CIEMAT is supported by grants from the European Commission (EUROFANCOLEN; ES2012-305421), Ministerio de Econom?a y Competitividad (SAF2015-68073), Fondo de Investigaciones Sanitarias (ISCIII;RD12/0019/0023; EC11/559 and 560), and from Rocket Pharma. Authors would also like to acknowledge the invaluable support from the Fanconi Anemia Research Fund, the Spanish Fanconi Anemia Foundation, and the Fanconi Hope Foundation, as well as from FA patients and families
- Fanconi anemia
- Gene editing
- Gene therapy
- Induced pluripotent stem cells