Investigating Therapeutic Targets for Myeloid Leukemia Using Gene Editing

Professor Introduction

S. Z | Ph.D. in  Cell Biology

Home Institute：Peking University

Project Description

Chronic myeloid leukemia (CML) is a common hematological disease, traditionally treated through bone marrow matching, which has a low success rate. The occurrence of leukemia is often associated with mutations or overexpression of oncogenes, but the regulatory mechanisms and therapeutic prospects remain unclear. The recent advancements in gene editing provide powerful tools for screening therapeutic targets for leukemia. This project aims to use gene editing tools to knock out a series of oncogenes in CML cells, with the goal of inhibiting leukemia cell proliferation and activating differentiation gene expression. First, we will construct gene knockout cells; next, we will measure their malignant proliferation and differentiation capabilities in vitro; finally, we will validate the in vivo efficacy in mouse models. This project could provide effective target references for the clinical treatment of myeloid leukemia.

Project Keywords

Project Outline

Part 1 : Introduction to  Chronic Myeloid Leukemia and Gene Editing
• Overview of CML and its current treatment challenges
• Importance of identifying new therapeutic targets using gene editing

Part 2: Research Objectives and Hypotheses
• Investigating the impact of oncogene knockout on CML cell proliferation and differentiation
• Exploring the therapeutic potential of gene-edited CML cells

Part 3: Review of Existing Research and Technologies
• Current understanding of oncogenes in CML and their roles in disease progression
• Overview of gene editing technologies and their applications in cancer research

Part 4:  Experimental Design and Gene Editing Techniques
• Designing experiments for gene knockout in CML cells using CRISPR-Cas9
• Techniques for constructing gene knockout cell lines

Part 5:   In Vitro Analysis of Gene-Edited CML Cells
• Methods for measuring cell proliferation and differentiation in gene-edited CML cells
• Analyzing the effects of oncogene knockout on cell behavior

Part 6:  In Vivo Validation in Mouse Models
• Setting up mouse models to validate the in vivo efficacy of gene-edited CML cells
• Evaluating the therapeutic potential and safety of gene-edited cells in vivo

Part 7:   Data Analysis and Interpretation
• Utilizing bioinformatics tools to analyze experimental data
• Identifying key genes and pathways affected by gene editing

Part 8: Development of Potential Therapeutic Strategies
• Exploring the feasibility of translating gene editing findings into clinical applications
• Evaluating the benefits and challenges of proposed therapeutic strategies

Part 9: Potential Applications in CML Treatment
• Discussing the therapeutic potential of identified targets
• Evaluating the benefits and challenges of implementing these strategies in clinical settings

Part 10: Results and Discussion
• Presenting experimental and computational results
• Discussing the significance and potential applications of the research findings
• Comparing new therapeutic targets with existing CML treatments

Part 11: Conclusion and Future Research Directions
• Summarizing key findings and their importance
• Identifying research limitations and suggesting future research directions
• Proposing practical applications of gene editing in various fields

Part 12: Reporting and Presentation
• Writing a comprehensive research report with clear structure, concise language, and accurate data presentation
• Preparing and delivering an engaging oral presentation of the research background, methods, results, and conclusions

Suitable for

High School Students:  
• Interest in Biomedical Research: Students strongly interested in biomedical engineering and gene editing technologies.
• Basic Knowledge:  Students with basic knowledge of biology, genetics, and molecular biology techniques.

University Students:
• Relevant Major:   Students majoring in biomedical engineering, molecular biology, genetics, or related fields.
• Technical Skills:  Familiarity with gene editing tools (e.g., CRISPR-Cas9), cell culture techniques, and molecular biology methods.

Researchers and Educators:
• Advanced Knowledge: Professionals with deep knowledge in hematology, gene editing, and cancer biology.
• Teaching Integration:  Ability to integrate research findings and methods into their courses and research work.