诱导多能性和基因编辑用于Fanconi贫血

Title:Induced Pluripotency and Gene Editing in Fanconi Anemia

Volume: 16 Issue: 5

关键词: 诱导多能干细胞，基因编辑，范可尼贫血，基因治疗。

摘要: 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.

Cite this article as:

Induced Pluripotency and Gene Editing in Fanconi Anemia, Current Gene Therapy 2016; 16 (5) . https://dx.doi.org/10.2174/1566523217666170118112050