Advanced Gene Editing of iPSCs Using CRISPR/Cas9
Unlocking the Potential of iPSCs—CRISPR/Cas9 for Precise Gene Editing and Disease Modeling
Skills you will gain:
About Program:
Gene editing using CRISPR/Cas9 has become a revolutionary tool in genetic research, enabling precise modifications at the genome level. Induced pluripotent stem cells (iPSCs), generated from adult cells, hold immense potential for regenerative medicine, disease modeling, and drug discovery. CRISPR/Cas9 allows for the precise targeting and modification of specific genes within iPSCs, enabling the creation of disease models and providing insights into the genetic basis of diseases like neurodegenerative disorders, cancer, and genetic syndromes.
This workshop covers the essential principles of CRISPR/Cas9 technology, including gRNA design, delivery methods, genomic target validation, and troubleshooting strategies for successful editing in iPSCs. Participants will gain practical insights through dry-lab exercises, including editing techniques, clone generation, and analysis of editing efficiency and off-target effects. The workshop also covers the potential applications of gene-edited iPSCs in disease modeling, regenerative medicine, and drug screening.
Aim: This workshop aims to provide participants with a comprehensive understanding of CRISPR/Cas9 technology for gene editing in induced pluripotent stem cells (iPSCs). It focuses on the principles and techniques of CRISPR/Cas9, from design and delivery to targeted genome modifications in iPSCs, and how this technology is used to model diseases, understand genetic functions, and develop therapeutic applications. The program bridges stem cell biology, genetic engineering, and gene editing techniques for translational research.
Program Objectives:
Learn the fundamental principles of CRISPR/Cas9 gene editing technology.1`q
Gain expertise in designing gRNAs for targeted gene modification in iPSCs.
Explore delivery methods and techniques for introducing CRISPR/Cas9 into iPSCs.
Understand the efficiency and validation of gene editing in iPSCs, including off-target effects.
Apply gene-edited iPSCs to disease modeling, therapeutic research, and drug screening.
What you will learn?
Day 1: Foundations of Genome Editing Technologies
- Overview of gene editing tools
- Evolution of genome editing: ZFN, TALENs, and CRISPR/Cas9
- Mechanisms behind CRISPR/Cas9 gene editing
- Understanding guide RNA and Cas9 interaction
- Overview of bacterial endonucleases and their role in target recognition
- Overview of DNA repair pathways: NHEJ vs HDR
- Manipulating repair pathways to enhance CRISPR/Cas9 outcomes
- Designing efficient experiments with CRISPR/Cas9 for iPS cells
Day 2: Advanced Applications of CRISPR/Cas9
- Medical applications: gene therapies, cancer research, and more
- CRISPR in agriculture and environmental science
- Distinct gene editing patterns in different bacterial systems
- Evolution of CRISPR systems across species
- Utilizing CRISPR/Cas9 libraries for genome-wide screens
- In vitro and in vivo screening techniques
CRISPR/Cas9 applications in stem cell reprogramming - Hands On: CRISPR Design Tools (e.g., Benchling, CRISPRdirect)
- Hands On: Gene editing in iPS cells with CRISPR
- Hands On: In vivo screening with CRISPR libraries (e.g., GeCKO libraries)
Day 3: Advanced Techniques and Applications in Genome Research
- Developmental biology: Using CRISPR for gene function analysis
- Potential for gene therapies in clinical settings
- CRISPR/Cas13 for RNA targeting and regulation
- Applications in transcriptomics and gene expression regulation
- Gene therapies in treating genetic disorders
- CRISPR’s role in precision medicine and ethical considerations
- Overcoming off-target effects and improving precision
- The future of CRISPR in gene editing for therapy
- Hands On: CRISPR/Cas13 RNA targeting tools (e.g., RNA Sniping)
- Hands On: Application of CRISPR in developmental biology
- Hands On: Gene Therapy Simulation Tools for CRISPR-based therapies
Mentor Profile
Fee Plan
Get an e-Certificate of Participation!
Intended For :
Undergraduate/postgraduate degree in Biotechnology, Molecular Biology, Cell Biology, Genetics, Bioinformatics, or related fields.
Professionals working in stem cell research, gene therapy, or biotechnology R&D sectors.
Genetic engineering and CRISPR/Cas9 enthusiasts interested in applying these techniques to iPSCs.
Individuals with a keen interest in regenerative medicine, disease modeling, and therapeutic development.
Career Supporting Skills
Program Outcomes
Understand and implement CRISPR/Cas9 techniques for iPSC gene editing.
Apply gRNA design and optimize gene modification in iPSCs.
Analyze editing efficiency and off-target effects in iPSC applications.
Develop iPSC-based disease models for therapeutic research.
Gain insights into the future applications of gene-edited iPSCs in regenerative medicine.