UNIQUE SERIES AKTU Syllabus
BBI061

Gene Therapy

L-T-P3-0-0 Credits3 Session2026-27 TypeTheory
Appears in 1 semester

B.Tech Bioinformatics · Sem 6 (Departmental Elective-III)

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Syllabus

5 units · 40 lecture hours
Unit1

Unit 1: GENERAL PRINCIPLES OF GENE THERAPY

9 hrs

Diseases that could be treated by gene therapy- Clinical trials- Gene Delivery. Reporter genes and Transfer efficiency- Germline vs. somatic cells- In vivo and ex vivo gene therapy- Ex vivo and In vivo Gene Therapy.

Unit2

Unit 2: DNA–BASED GENE THERAPY

7 hrs

Mechanical methods of delivery Example: Duchenne myotrophy- Liposomal methods of delivery- Cystic fibrosis.

Unit3

Unit 3: ADENO-ASSOCIATED VIRUS-BASED VECTORS

8 hrs

Safety of GT vectors- Gutted vectors- Helper viruses and Packaging cell lines- Examples: ornithine transcarbamylase deficiency (OTC) - Severe Combined Immunodeficiency (SCID). -Failures in the OTC and SCID GT trials.

Unit4

Unit 4: EVENT HANDLINGS AND AWT CONTROLS

8 hrs

Hemophilia Diabetes mellitus- Erythropoietin- How to target vector to the particular cell- Tissue specific promoters- Vectors as ligands for cellular receptors.

Unit5

Unit 5: CANCER GENE THERAPY

8 hrs

Genetic Prodrug Activation Gene Therapy- GT methods to overcome chemotherapy resistance of tumors- Biological response modifiers- Oncolytic virus-based strategies of tumor treatment. DNA vaccines for cancer and infectious diseases- AIDS as a target for DNA vaccines- Antisense based methods of gene therapy.

Course Outcomes (COs)5 outcomes

After completion of the course student will be able to:

  1. CO1Understand general principles of gene therapy, delivery methods, and clinical trial approaches.K2
  2. CO2Apply DNA-based gene therapy techniques including mechanical and liposomal delivery.K3
  3. CO3Explain retroviral and adenoviral vectors, their safety, and case studies (OTC, SCID).K2
  4. CO4Explain adeno-associated virus vectors, tissue-specific promoters, and targeted delivery strategies.K2
  5. CO5Describe cancer gene therapy methods including prodrug activation, oncolytic viruses, DNA vaccines, and antisense approaches.K2

TEXT BOOKS

  1. / REFERENCES Dr. A.P.J. Abdul Kalam Technical University, Lucknow, Uttar Pradesh
  2. Peter J. Quesenberry, “Stem cells biology and gene therapy”, Wiley-Liss, 1998.
  3. Keith Greenberg, “Gene therapy”, Blackbirch Pr Inc, 2003. Dr. A.P.J. Abdul Kalam Technical University, Lucknow, Uttar Pradesh

Official AKTU syllabus

This subject is on page 35, 36, 37 of the original AKTU PDF.

Official AKTU syllabus PDF ↗