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Therapeutic gene editing in haematological disorders with CRISPR /Cas9

2019; Wiley; Volume: 185; Issue: 5 Linguagem: Inglês

10.1111/bjh.15851

1365-2141

Trine I. Jensen, Esben Axelgaard, Rasmus O. Bak,

Pluripotent Stem Cells Research

Summary The Clustered Regularly Interspaced Short Palindromic Repeats ( CRISPR )/ CRISPR ‐associated (Cas)9 platform offers an efficient way of making precise genetic changes to the human genome. This can be employed for disruption, addition and correction of genes, thereby enabling a new class of genetic therapies that can be applied to haematological disorders. Here we review recent technological advances in the CRISPR /Cas9 methodology and applications in haematology for curing monogenic genetic disorders and for engineering novel chimeric antigen receptor ( CAR ) T cells to treat haematological malignancies. Furthermore, we discuss current challenges for full clinical implementation of CRISPR /Cas9, and reflect on future trajectories of the technology.