Therapeutic gene editing in haematological disorders with CRISPR/Cas9

@article{Jensen2019TherapeuticGE,
  title={Therapeutic gene editing in haematological disorders with CRISPR/Cas9},
  author={Trine I Jensen and Esben Axelgaard and Rasmus O. Bak},
  journal={British Journal of Haematology},
  year={2019},
  volume={185}
}
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… 

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