DISEASE MODELING AND THERAPEUTICS

Registration and Welcome

Opening Lecture

iPSC-Based Disease Modeling: A New Era in Translational Research

An overview of the role of iPSC models in human disease research and personalized medicine.

Lecture

Essentials of iPSC Technology: From Reprogramming to Quality Control

A foundational introduction to iPSC biology, reprogramming strategies, and quality assessment.

Coffee Break

Lecture

Introduction to CRISPR/Cas9 Genome Editing in iPSCs

A practical guide to implementing CRISPR/Cas9 in stem cell models

Practical Session I: iPSC Culture & Maintenance

Format: Hands-on, small-group rotation.

This session provides a technical foundation for researchers working with or planning to establish iPSC cultures. Through guided, hands-on activities at dedicated lab stations, participants will gain essential skills in the cultivation and maintenance of high-quality iPSC lines. Participants will work closely with instructors to observe and practice key techniques, including colony assessment, passaging, and cell banking.

Lunch Break

Practical Session II: Reprogramming Somatic Cells to iPSCs

Format: Hands-on, small-group rotation.

This session offers participants a practical introduction to reprogramming somatic cells into iPSCs, with a focus on peripheral blood mononuclear cells (PBMCs). Participants will observe and perform key steps in the reprogramming workflow, including PBMC expansion, viral transfection, and colony selection.

By the end of this session, participants will gain hands-on experience and practical insights into optimizing reprogramming efficiency and establishing high-quality iPSC lines.

Practical Session III: Genome Editing with CRISPR/Cas9

Format: Hands-on demonstration and guided discussion

This session offers an in-depth introduction to genome editing in human iPSCs using the CRISPR/Cas9 system. Participants will observe a step-by-step demonstration covering the full workflow, including guide RNA (gRNA) design and transfection techniques.

The session will conclude with a group discussion focused on validation strategies and methods for identifying successfully edited clones.

By the end of the session, participants will gain practical insights into optimizing editing efficiency, troubleshooting common challenges, and implementing best practices for CRISPR-based genome editing in iPSC models.

Wrap-Up Discussion & Q&A

Practical Session IV: Follow-Up Practical Checkpoints

Format: Hands-on, small-group rotation.

In this session, participants will revisit key steps from previous practical sessions, with a focus on evaluating progress and addressing any challenges. This session provides an opportunity for hands-on troubleshooting, with guidance from instructors to reinforce best practices and refine techniques.

Lecture

Modeling Diabetes with iPSC-Derived Lineages

Applications of patient-specific and gene edited iPSC models in different types of diabetes

Coffee Break

Lecture

Directed Differentiation of iPSCs into Pancreatic Islets

Overview of the stepwise differentiation protocols and key checkpoints

Lecture

Differentiation of iPSCs into Hepatic Organoids

Generation of functional hepatocytes for modeling liver-related diseases

Lecture

iPSC Models in Neurodevelopmental and Neurodegenerative Disorders

Exploring cortical neuron differentiation and 3D brain organoid systems

Practical Session V: Pancreatic Differentiation

Format: Hands-on demonstration and guided practice.

In this session, participants will explore the essential steps involved in differentiating iPSCs into pancreatic cells. They will follow a stepwise protocol to induce pancreatic differentiation and observe the formation of 3D pancreatic islet organoids. By the end of this session, participants will have acquired hands-on skills in pancreatic differentiation and the functional assessment of iPSC-derived insulin-secreting beta cells.

Lunch Break

Parallel Practical Sessions

Session VI: Hepatic Differentiation from iPSCs

Format: Hands-on demonstration and guided practice

In this session, participants will explore the process of differentiating iPSCs into hepatic lineage cells. They will follow a protocol to induce hepatic differentiation and observe the generation of organoids. By the end of the session, participants will gain hands-on experience in hepatic differentiation and the functional assessment of iPSC-derived liver cells.

Session VII: Neuronal Differentiation from iPSCs

Format: Hands-on demonstration and guided practice

This session will guide participants through the generation of neural progenitors and cortical neurons from iPSCs. Participants will observe the formation of neural rosettes and neuronal organoids, while also exploring key markers and characterization strategies to assess neuronal differentiation. By the end of the session, participants will have acquired practical skills in neuronal differentiation and the characterization of iPSC-derived neuronal cells.