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Therapeutic gene editing in haematological disorders with CRISPR /Cas9

12 Mar 2019

Abstract excerpt

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...

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PMID: 30864164
DOI: 10.1111/bjh.15851

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