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CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

7 Jun 2020

Abstract excerpt

Genome editing therapies represent a significant advancement in next-generation, precision medicine for the management of haematological diseases, and CRISPR/Cas9 has to date been the most successful implementation platform. From discovery in bacteria and archaea over three decades ago, through intensive basic research and pre-clinical development phases involving the modification of therapeutically relevant cell...

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PMID: 32506752
DOI: 10.1111/bjh.16807

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