Therapeutic gene editing in haematological disorders with CRISPR/Cas9

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DOI

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.

Original languageEnglish
JournalBritish Journal of Haematology
Volume185
Issue5
Pages (from-to)821-835
Number of pages15
ISSN0007-1048
DOIs
Publication statusPublished - 2019

    Research areas

  • CAR, CRISPR, CRISPR-CAS9, Cas9, DNA, GENOME, IMMUNODEFICIENCY, INTERLEUKIN-15, MUTATION, REPOPULATING HEMATOPOIETIC STEM, RNA, T-CELLS, TARGET, haematology, monogenic

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