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Therapeutic gene editing in haematological disorders with CRISPR /Cas9
Author(s) -
Jensen Trine I.,
Axelgaard Esben,
Bak Rasmus O.
Publication year - 2019
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.15851
Subject(s) - crispr , genome editing , cas9 , chimeric antigen receptor , computational biology , gene , biology , bioinformatics , genetics , immunotherapy , cancer
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.