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Course Detail

Course Name Principles and Applications of Cell and Gene Therapy
Course Code 25SCR515
Program M. Sc. Stem Cell Technology and Regenerative Medicine
Semester 2
Credits 2
Campus Kochi

Syllabus

Module 1

7 Hrs

Fundamentals of Cell Therapy; Introduction to cell therapy, definitions, scope, and therapeutic promise; historical evolution of cell-based treatments and their impact; distinction and interplay between cell and gene therapies; overview of therapeutic cell sources (autologous vs. allogeneic); key cell types in therapy: stem cells (ESCs, iPSCs, adult stem cells), immune cells (CAR-T, NK, dendritic), and MSCs-highlighting clinical applications; regulatory classification of cell-based products, overview of translational bottlenecks and considerations for first-in-human use.

Module 2

8 Hrs

Applications and Challenges in Cell Therapy; hematopoietic stem cell transplantation, CAR-T cell therapy for cancer, use of MSCs in regenerative and inflammatory diseases; delivery methods: local vs. systemic administration, targeted delivery approaches; monitoring techniques: cell tracking, biodistribution, and efficacy assessment; safety challenges: tumorigenicity, immunogenicity, off-target effects; regulatory frameworks and approvals (FDA, EMA); ethical issues surrounding stem cell sourcing and manipulation.

Module 3

7 Hrs

Molecular and Technical Foundations of Gene Therapy; fundamentals of DNA, RNA, and gene expression; genetic diseases as targets for gene therapy; viral vectors: retrovirus, lentivirus, adenovirus, adeno-associated virus (AAV) – mechanisms, advantages, limitations; non-viral gene delivery methods: plasmids, liposomes, nanoparticles, electroporation, microinjection, sonoporation – safety and efficiency considerations; gene editing tools: CRISPR-Cas9, TALENs, zinc finger nucleases (ZFNs) – mechanisms and therapeutic applications.

Module 4

8 Hrs

Targeting, Strategies; transductional targeting, transcriptional targeting, inducible and tissue-specific promoters; gene inhibition and ablation strategies: antisense constructs, antisense oligonucleotides, ribozymes, intracellular antibodies, RNA interference (RNAi); safety concerns in preclinical and clinical gene therapy studies; ethical debates on somatic vs. germline editing; legal and regulatory considerations for gene therapy worldwide.

Introduction

Pre-requisites: Undergraduate level basic biology

Total number of classes: 30

Preamble

Cell and gene therapies are revolutionizing the treatment of genetic disorders, cancers, and degenerative diseases by offering targeted and durable interventions at the cellular and molecular levels. This course introduces the foundational principles, therapeutic applications, and translational challenges of both cell- and gene-based therapies. Emphasis is placed on clinically relevant cell types, gene delivery systems, genome editing tools, and associated ethical and regulatory frameworks. Designed for students with basic biology knowledge, the course bridges molecular mechanisms with emerging clinical strategies, fostering a deep understanding of next-generation biotherapeutics.

Objectives and Outcomes

Course Outcomes (COs)

CO1: Describe key concepts, sources, and regulatory considerations in cell therapy using stem and immune cells.

CO2: Assess clinical applications, delivery strategies, and challenges in translating cell-based therapies.

CO3: Explain the molecular basis of gene therapy, including vectors and genome editing tools.

CO4: Evaluate gene targeting strategies, and discuss safety, ethical, and legal issues in somatic and germline gene therapy.

PO1: Bioscience Knowledge

PO2: Problem Analysis

PO3: Design/Development of Solutions

PO4: Conduct Investigations of complex problems

PO5: Modern tools usage

PO6: Bioscientist and Society

PO7: Environment and Sustainability

PO8: Ethics

PO9: Individual & Team work

PO10: Communication

PO11: Project management & Finance

PO12: Lifelong learning

0 – No affinity; 1 – low affinity; 2 – Medium affinity; 3 – High affinity

2

PO1

PO2

PO3

PO4

PO5

PO6

PO7

PO8

PO9

PO10

PO11

PO12

C O

CO 1

3

2

1

1

1

–

1

3

1

2

–

3

CO 2

3

3

2

2

2

–

2

3

2

2

–

3

CO 3

3

2

2

2

3

–

1

2

1

1

–

3

CO 4

3

2

2

2

2

–

2

3

1

2

–

3

Program Specific Outcomes. (PSO)

PSO1. Demonstrate comprehensive knowledge of stem cell biology and their clinical and research relevance.

PSO2. Apply core laboratory techniques for stem cell isolation, characterization, and manipulation.

PSO3. Integrate principles of tissue engineering and biomaterials for regenerative applications.

PSO4. Analyze drug delivery, pharmacokinetics, and bioinformatics relevant to stem cell-based therapies.

PSO5. Evaluate ethical, regulatory, and translational aspects of stem cell product development.

PSO6. Bridge basic science with translational approaches in regenerative medicine and gene therapy.

PSO7. Design and interpret experimental strategies for stem cell-based disease modeling and preclinical studies.

–

PSO1

PSO2

PSO3

PSO4

PSO5

PSO6

PSO7

C O

CO 1

3

2

3

–

3

1

3

CO 2

3

–

3

–

2

2

2

CO 3

–

–

–

–

2

2

–

CO 4

–

–

–

–

–

3

–

Evaluation Pattern

Evaluation Pattern: 50+50 = 100

Internal Assessment – 50%
Periodical 1  Exam 20%
Periodical 2  Exam 20%
Continuous Assessment  Assignment/Test/Quiz 10%
50%
End Semester Examination- 50%
Theory Exam 50%
50%
Total 100%

Textbook/ Reference

  1. Aranha, Hazel, and Humberto Vega-Mercado, eds. 2023. Handbook of Cell and Gene Therapy: From Proof-of-Concept through Manufacturing to Commercialization. Elsevier.
  2. George Morstyn, William Sheridan , Amgen Inc. (2020). Cell therapy: Stem cell transplantation, gene therapy, and cellular immunotherapy
  3. Bonetti, A. (Ed.). (2010). Gene therapy. Springer.
  4. Brown, P. (2018). Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective. Informa Healthcare.

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