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Submitted by:
H. Bharath Raj(13191A2301)
 September 14, 1990 – Kum. Ashanti De
Silva(SCID).
 Approaches of Gene Therapy:
Somatic cell Gene Therapy and
Germ cell Gene Therapy
 4 steps are followed to develop G.T for any
specific disease
 “Recombinant DNA Advisory
Committee(RAC)”
 There are two types of gene therapy:
They are: “Ex vivo” and “In vivo” gene therapy.
 Ex vivo gene therapy: (steps)
 Vectors in Gene Therapy: (carrier particles)
a.) Viruses
b.) Human Artificial Chromosomes
c.) Bone marrow cells
 Retro-virus:
Harmful retro virus to
harmless retrovirus.
 Genetic map of Retrovirus: (organised in 6 regions)
 The other virus are:
Adeno virus, Adeno – associated virus and
Herpes Simplex Virus.
 AIDS virus in gene therapy:
(non-dividing cells like
brain cells effectively)
Human Artificial Chromosomes:
Synthetic chromosome,
Bone marrow cells:
 Contains totipotent embryonic stem cell.
 Diseases treated are,
 Examples of Ex-vivo gene therapy:
1.) SCID: (T-lymphocytes and B- lymphocytes)
 Therapy for it through Ex-vivo G.T
 2.) Therapy for Lesch – Nyhan syndrome:
 It is an inborn error in purine metabolism
 HGPRT (Retroviral vector system)
3.) Therapy for Haemophilia:
• Lack of clotting factor IX
• Retroviral vector system
In vivo Gene therapy
 Liver, muscle, skin, spleen, lung, brain and blood cells
Gene delivery by virus:
 Adenoviral vector system: expresses the protein by
itself, does not integrate with the DNA of host cell.
 Adeno Associated virus vector system: Diseases treated
are Hemophilia, Cystic Fibrosis(CF)
 Therapy for Cystic Fibrosis: sticky, dehydrated
mucous; CFTR, pushes Cl ions
 Gene therapy:
 Adeno viral vector system
 Adeno associated
virus vector system
 Herpes Simplex virus vector system: treats the
nervous didorders; Alzheimers, Parkinson’s
disease (if successful)
 Limitation: Genome length.
Gene delivery by non-viral vector
system:
 Pure DNA constructs:
 Simple; frequently degraded
Lipoplexes:
But the benefits are short lived.
Human Artificial Chromosomes:
Can carry one or more therapeutic genes.
 Gene therapy for AIDS:
‘rev’ and ‘env’ genes
Antigen and antisense therapy:
 Transcription using
a single stranded
nucleotide sequence.
 Antisense therapy:
Conclusion:
 Permanent solution
 Accessible
 Breakthrough
 Revolutionise the practice of medicine
References:
 The text book of Bio Technology by
Dr. U. Satyanarayana.
 www.slideshare.net
 www.youtube.com
 Google images.
Queries:
Thank you…

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Gene therapy

  • 1. Submitted by: H. Bharath Raj(13191A2301)
  • 2.  September 14, 1990 – Kum. Ashanti De Silva(SCID).  Approaches of Gene Therapy: Somatic cell Gene Therapy and Germ cell Gene Therapy  4 steps are followed to develop G.T for any specific disease
  • 3.  “Recombinant DNA Advisory Committee(RAC)”  There are two types of gene therapy: They are: “Ex vivo” and “In vivo” gene therapy.  Ex vivo gene therapy: (steps)
  • 4.  Vectors in Gene Therapy: (carrier particles) a.) Viruses b.) Human Artificial Chromosomes c.) Bone marrow cells
  • 5.  Retro-virus: Harmful retro virus to harmless retrovirus.  Genetic map of Retrovirus: (organised in 6 regions)
  • 6.  The other virus are: Adeno virus, Adeno – associated virus and Herpes Simplex Virus.  AIDS virus in gene therapy: (non-dividing cells like brain cells effectively)
  • 7. Human Artificial Chromosomes: Synthetic chromosome, Bone marrow cells:  Contains totipotent embryonic stem cell.  Diseases treated are,
  • 8.  Examples of Ex-vivo gene therapy: 1.) SCID: (T-lymphocytes and B- lymphocytes)  Therapy for it through Ex-vivo G.T
  • 9.  2.) Therapy for Lesch – Nyhan syndrome:  It is an inborn error in purine metabolism  HGPRT (Retroviral vector system) 3.) Therapy for Haemophilia: • Lack of clotting factor IX • Retroviral vector system
  • 10. In vivo Gene therapy  Liver, muscle, skin, spleen, lung, brain and blood cells Gene delivery by virus:  Adenoviral vector system: expresses the protein by itself, does not integrate with the DNA of host cell.  Adeno Associated virus vector system: Diseases treated are Hemophilia, Cystic Fibrosis(CF)
  • 11.  Therapy for Cystic Fibrosis: sticky, dehydrated mucous; CFTR, pushes Cl ions  Gene therapy:  Adeno viral vector system  Adeno associated virus vector system  Herpes Simplex virus vector system: treats the nervous didorders; Alzheimers, Parkinson’s disease (if successful)  Limitation: Genome length.
  • 12. Gene delivery by non-viral vector system:  Pure DNA constructs:  Simple; frequently degraded Lipoplexes: But the benefits are short lived. Human Artificial Chromosomes: Can carry one or more therapeutic genes.
  • 13.  Gene therapy for AIDS: ‘rev’ and ‘env’ genes Antigen and antisense therapy:  Transcription using a single stranded nucleotide sequence.  Antisense therapy:
  • 14. Conclusion:  Permanent solution  Accessible  Breakthrough  Revolutionise the practice of medicine
  • 15. References:  The text book of Bio Technology by Dr. U. Satyanarayana.  www.slideshare.net  www.youtube.com  Google images. Queries: