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Updated: Jan 27, 2026

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
Published on: June 20, 2019
Therapeutic gene editing in haematological disorders with CRISPR/Cas9
Trine I Jensen1, Esben Axelgaard1, Rasmus O Bak1,2
1Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
Abstract:
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.
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