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Updated: Nov 16, 2025
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Mini review: genome and transcriptome editing using CRISPR-cas systems for haematological malignancy gene therapy
Esther K Elliott1, Larisa M Haupt1, Lyn R Griffiths2
1Centre for Genomics and Personalised Health, Genomics Research Centre, School of Biomedical Sciences, Institute of Health and Biomedical Innovation, Queensland University of Technology (QUT), 60 Musk Ave, Kelvin Grove, QLD, 4059, Australia.
CRISPR-Cas13 systems offer novel RNA editing tools for transcriptome engineering and gene expression modification. These systems show promise for cancer therapy, disease modeling, and diagnostics in hematological malignancies.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR associated protein (Cas) systems provide genome and transcriptome editing capabilities.
- CRISPR-Cas13 effectors specifically target RNA, enabling transcriptome engineering and gene expression modulation.
Purpose of the Study:
- To review adaptations of CRISPR-Cas systems for RNA modification.
- To explore applications in disease modeling and gene therapy for hematological malignancies.
- To assess the diagnostic and therapeutic potential of CRISPR-Cas systems in personalized hematological malignancy treatment.
Main Methods:
- Review of existing literature on CRISPR-Cas systems, focusing on Cas13 effectors.
- Analysis of RNA base editing techniques (e.g., A to I replacement) using Cas13.
- Examination of Cas13a applications in mobile diagnostic platforms.
Main Results:
- CRISPR-Cas13 systems enable precise transcript modification and gene expression control.
- Cas13a facilitates rapid diagnostics on mobile platforms.
- CRISPR-Cas systems are adaptable for transcriptome modifications in disease modeling and gene therapy.
Conclusions:
- CRISPR-Cas13 systems represent a significant advancement in RNA-based therapeutic strategies.
- These systems hold potential for personalized treatments and diagnostics in hematological malignancies.
- Further research into CRISPR-Cas applications can advance cancer therapy and disease management.
