Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Induced Pluripotency and Gene Editing in Fanconi Anemia

Author(s): Susana Navarro, Alessandra Giorgetti, Angel Raya, Jakub Tolar.

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.

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Article Details

VOLUME: 16
ISSUE: 5
Page: [321 - 328]
Pages: 8
DOI: 10.2174/1566523217666170118112050
Price: $58