CRISPR-Based Gene Editing in Haematological Malignancies: Current Evidence and Translational Barriers
Division of Molecular Genetics and Department of Haematology, Institute of Biomedical Sciences, Kolkata, West Bengal, India
Corresponding Author: Das M, Doctus Journal of Biomedical Sciences.
Abstract
Genome editing has moved from proof-of-concept to registered clinical trials in haematological malignancies within a single decade. This review synthesises 96 preclinical reports and 21 registered trials published between 2019 and 2026, covering CAR-T manufacture, base editing of transcription factor binding sites, and ex-vivo correction of haemoglobinopathies. Editing efficiency in primary human haematopoietic stem cells now routinely exceeds 70%, but three barriers dominate the translational gap: off-target activity that current detection assays measure inconsistently, the cost and cold-chain burden of autologous manufacture, and the absence of harmonised regulatory endpoints across jurisdictions. We propose a minimum reporting standard for off-target analysis and argue that allogeneic, edited donor products offer the most realistic route to scale in resource-constrained health systems.
Keywords
CRISPR; Gene Editing; Haematological Malignancy; CAR-T; Base Editing; Translational Research
How to Cite
Das M, Ghosh K and Bhattacharya T. “CRISPR-Based Gene Editing in Haematological Malignancies: Current Evidence and Translational Barriers”. Doctus Journal of Biomedical Sciences 2.11 (2026): 44-63.
Copyright and Licence
© 2026 Das M. This is an open access article distributed under the Creative Commons Attribution Licence, which permits unrestricted use, distribution and reproduction in any medium, provided the original work is properly cited.