Course Offerings

Cell and Gene Therapy

Course ID: BTC-3300H6
Overview
Course Outline
Requirements
Intended Audience
More Details

This introductory course for cell and gene therapy is designed to give participants the basic knowledge and the strategies that can be used to create products from molecular biotechnology techniques. It also aims to give them a background into genetics, cell culture techniques, and gene sequencing so that there can be understanding how these concepts play into industrial processes to make useful therapeutic products. Ethical and regulatory considerations are also presented.

Course Objectives

Learn the basics of microbiology, virology, and immunology.
Be exposed to various molecular biology techniques, including DNA/RNA extraction, PCR techniques, genotyping, sequencing, gel electrophoresis, and more.
Learn how these molecular cell and gene therapy techniques can be used in applications for commercial product manufacturing.
Learn the basics of the process from inception in research and development through product realization in manufacturing.

Outline of Instruction

1. Cell Biology
a. Eukaryotic vs prokaryotic
b. Structures and functions
2. Microbiology
3. Virology
a. Characteristics
b. Types
4. Immunology
a. Immunotherapy
5. Genetics
a. DNA vs RNA
b. Mitosis vs Meiosis
c. Central Dogma and its steps
i. Video (From DNA to Protein)
d. Genes/Genotype/Phenotype
i. Human Genome Sequencing Video
ii. Variations (Sequencing.com Interactive Genome Map)
iii. Genetic Disorders
6. Concepts of Molecular Biology and Biotechnology
a. DNA Extraction/ RNA Extraction
b. PCR
c. Genotyping vs Sequencing
d. Gel Electrophoresis
e. Gene expression
i. Regulation
f. Gene Editing
i. CRISPR/Cas9
g. Recombinant DNA
h. Plasmid Construction
i. Insulin Production Example
i. Cell Culture
i. Transfection
ii. Transformation
iii. Transduction
iv. Applications
j. Aseptic Technique
7. Cell vs Gene Therapy
a. Gattaca Video
b. Applications and Products
c. Types
i. Somatic
ii. Germline
iii. In Vivo
iv. Ex Vivo
d. Techniques
i. Augmentation
ii. Inhibition
iii. Suicidal
e. Delivery
i. Viral
ii. Non-Viral
f. Case studies
8. Development of drug process
a. Discovery, Development, and Launch Process
b. Comparison to regular biomanufacturing
9. FDA regulation
a. Analytical Techniques
b. Product Safety Requirements
c. Cell Characterization
10. Ethical Concerns
11. CGMP
12. Good Documentation Practices