Related Experiment Video
Updated: Mar 22, 2026
Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells
Published on: September 1, 2019
Current attempts to implement siRNA-based RNAi in leukemia models
Hasan Uludağ1, Breanne Landry2, Juliana Valencia-Serna3
1Department of Chemical & Materials Engineering, Faculty of Engineering, University of Alberta, Edmonton, Canada; Department of Biomedical Engineering, Faculty of Medicine & Dentistry, University of Alberta, Edmonton, Canada; Faculty of Pharmacy & Pharmaceutical Sciences, University of Alberta, Edmonton, Canada.
Abstract:
Leukemias arise from genetic alterations in normal hematopoietic stem or progenitor cells, leading to abnormal blood population with transformed cells. With the advent of RNAi and its pharmacological mediator siRNA, it has become possible to downregulate specific drivers causing leukemias. In this review, we present unique aspects of RNAi-mediated therapy and delivery technologies. Recent updates on molecular targets and delivery systems are discussed emanating from in vitro cell models and preclinical animal models. We conclude with a view on the future of RNAi in leukemia therapy, emphasizing possible measures to achieve higher efficacy and improved safety.
Insights
RNAi therapy, using small interfering RNA (siRNA), offers a new way to target leukemia-causing genes. This review covers RNAi delivery methods and future strategies for more effective and safer leukemia treatments.
Area of Science:
- Hematology
- Molecular Biology
- Pharmacology
Background:
- Leukemias originate from genetic mutations in hematopoietic stem/progenitor cells.
- These mutations result in an overpopulation of abnormal, transformed blood cells.
- RNA interference (RNAi) offers a mechanism to silence specific disease-driving genes.
Purpose of the Study:
- To review RNAi-mediated therapy for leukemia.
- To discuss novel delivery technologies for RNAi.
- To explore future directions for enhanced efficacy and safety.
Main Methods:
- Review of current literature on RNAi therapy in leukemia.
- Analysis of in vitro and preclinical animal models.
- Examination of molecular targets and delivery systems for RNAi.
Main Results:
- RNAi, particularly with small interfering RNA (siRNA), can effectively downregulate specific oncogenic drivers.
- Various delivery technologies are being developed to enhance RNAi efficacy.
- Preclinical data support the potential of RNAi-based approaches.
Conclusions:
- RNAi-based therapies show promise for treating leukemias.
- Advancements in delivery systems are crucial for clinical translation.
- Future research should focus on optimizing efficacy and safety profiles.
Related Concept Videos
01:23RNA Interference
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
01:23RNA Interference
02:15Experimental RNAi
02:30siRNA - Small Interfering RNAs
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...