Induced Pluripotency and Gene Editing in Fanconi Anemia
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.
Keywords: Induced pluripotent stem cells, Gene editing, Fanconi anemia, Gene therapy.
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