Abstract
In recent years, tremendous advances have been made in the use of gene editing to precisely engineer the genome. This technology relies on the activity of a wide range of nuclease platforms — such as zinc-finger nucleases, transcription activator-like effector nucleases, and the CRISPR–Cas system — that can cleave and repair specific DNA regions, providing a unique and flexible tool to study gene function and correct disease-causing mutations. Preclinical studies using gene editing to tackle genetic and infectious diseases have highlighted the therapeutic potential of this technology. This review summarizes the progresses made towards the development of gene editing tools for the treatment of haematological disorders and the hurdles that need to be overcome to achieve clinical success.
Highlights
Genome engineering has endowed the scientific community with the ability to artificially modify genetic information, unlocking the potential of traditional medicine to new therapeutic approaches
M.R. prepared the figures under the supervision of A.C
Summary
Genome engineering has endowed the scientific community with the ability to artificially modify genetic information, unlocking the potential of traditional medicine to new therapeutic approaches. Gene correction might seem the most immediate approach to therapeutic genome editing, the first clinical trial using targeted nucleases in human patients has relied on NHEJ-based genetic disruption One advantage of this strategy is that NHEJ tends to be a more active repair pathway compared with HDR, in quiescent stem cells [28]. Can this technique allow the functional study of a particular gene, but it is amenable to correction of a disease-associated mutation Despite this exciting prospect, there remain major barriers associated with the editing-based therapeutic treatment of blood disorders that must be addressed to advance clinical applications that rely on genome editing. The continued improvements of the efficiency and safety of these reagents will be essential to ensure the success of gene editing applications to treat genetic haematological disorders
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