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.
Keywords: Induced pluripotent stem cells, Gene editing, Fanconi anemia, Gene therapy.
Current Gene Therapy
Title:Induced Pluripotency and Gene Editing in Fanconi Anemia
Volume: 16 Issue: 5
Author(s): Susana Navarro, Alessandra Giorgetti, Angel Raya and Jakub Tolar
Affiliation:
Keywords: Induced pluripotent stem cells, Gene editing, Fanconi anemia, Gene therapy.
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.
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Navarro Susana, Giorgetti Alessandra, Raya Angel and Tolar Jakub, Induced Pluripotency and Gene Editing in Fanconi Anemia, Current Gene Therapy 2016; 16 (5) . https://dx.doi.org/10.2174/1566523217666170118112050
DOI https://dx.doi.org/10.2174/1566523217666170118112050 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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