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Course Co-ordinated by IIT Guwahati
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Dr. Sachin Kumar
IIT Guwahati

 

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Introduction, Somatic and germ line gene therapy, Gene replacement and gene addition, In vivo, ex vivo and in vitro gene therapy, Transgenic animal models, Vehicles for gene transfer-viral vectors: retrovirus, Adenovirus, Adeno-associated virus, Lentivirus, Recombinant SV40 virus, Nonviral vectors, DNA vaccines, Liposomes and lipoplexes, Naked DNA, transposon, Cancer gene therapy, RNA-DNA chimera, Gene therapies for Criglar-Najjar syndrome I, Cystic fibrosis, Duchnne muscular dystrophy, Bleeding disorders, Tyrosinemia, Severe combined immunodeficiency syndrome (SCID), Gene therapy of non-heritable disorders, Recent advancement in Gene Therapy.

 
 

S. No.

Module

Topics and Contents

No of Lectures

1.

Introduction

Introduction, Somatic and germ line gene therapy, Gene replacement and gene addition.

6

2.

In vivo gene therapy

In vivo, ex vivo and in vitro gene therapy, Transgenic animal models, Vehicles for gene transfer-viral vectors: retrovirus, Adenovirus, Adeno-associated virus.

8

3.

Viral vectors

Lentivirus, Recombinant SV40 virus, Nonviral vectors, DNA vaccines, Liposomes and lipoplexes, Naked DNA, transposon.

8

4.

Cancer gene therapy

Cancer gene therapy, RNA-DNA chimera, Gene therapies for Criglar-Najjar syndrome I.

6

5.

Gene Therapy and diseases-I

Cystic fibrosis, Duchnne muscular dystrophy, Bleeding disorders, Tyrosinemia.

6

6.

Gene Therapy and diseases-II

Severe combined immunodeficiency syndrome (SCID), Gene therapy of non-heritable disorders, Recent advancement in Gene Therapy.

6

 

Total

40

  • Friedman T. 1999. The Development of Human Gene Therapy. Cold Spring Harbor,NY: Cold Spring Harbor Lab. Press.
  • Knipe DM, Howley PM, eds. 2001. Fields Virology. Philadelphia, PA: Lippincott Williams & Wilkins.
  • Hackett NR, Crystal RG. 2000. Adenovirus vectors for gene therapy. In Gene Therapy, ed. NS Templeton, DD Lasic, pp.17-39. New York: Marcel Dekker


  • Gene therapy: Twenty-First Century Medicine. Annu. Rev. Biochem. 2005. 74:711-38
  • Gene therapy: Promises and Problems. Annu. Rev. Genomics Hum. Genet. 2001. 2:177-211


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