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Viral Gene Therapy
Presented by Alex Bunk
What It Is
 Viral Gene therapy is the methodic use of viruses that
  normally cause illness as vectors to transfer genes that will
  produce desired effects in patients afflicted with certain
  illnesses.
What It Is
There is a lack of an efficient non-toxic delivery system to provide the
 successful application of gene therapy for treatment of a range of
 diseases has been a major barrier to gene therapy.

Viruses have evolved to deliver their genes to target cells and are
  considered to be the most effective means of gene delivery and have
  an ability to be manipulated to express therapeutic genes or to replicate
  specifically in certain cells. Gene therapy is being developed for a range
  of diseases including inherited monogenic disorders and cardiovascular
  disease, but it is in the treatment of cancer that this approach has been
  most evident, resulting in the licensing of a gene therapy for the routine
  treatment of head and neck cancer in China.
How It Works
 Viral gene therapy works by first removing the genes of a
  viral vector then replacing it with genes that will produce the
  desired effect.
What It Can Cure
 Viral gene therapy is capable of curing a multitude of
  diseases, both genetic and acquired.
 Diseases that viral gene therapy can cure include:
     Cancer
     Type 1 diabetes
     Sever combined immune deficiency
     Chronic granulomatous disorder
     Hemophilia
     Various cancers
     Neurodegenerative diseases
     Cystic fibrosis
Viral Vectors Used
 The two most commonly used vectors in the process are:
   Retroviruses
   Adenoviruses
 Other less frequently used viruses are:
     Adeno-associated viruses
     Lentiviruses
     Pox viruses
     Alphaviruses
     Herpes viruses
 These viruses all differ in how well they are able to transfer
  genes to the cells they recognize and are able to infect, and
  whether they alter the cell’s DNA temporarily or permanently
 These viruses are displayed on the following slide.
Risks
 There are a number of risk factors involved in viral gene therapy.
 Such problems include:
   Destruction of healthy cells in cancer treatment
   Cancerous mutations due to injection at incorrect locations on DNA
   Changes can be passed on to children if the reproductive cells are
    affected.
 In addition, interference from corporations that produce drugs
  for people with chronic illnesses, such as Medtronics who
  produce insulin pumps. Viral gene therapy is not supported by
  the entities that fund research because it would be the cure to
  the very diseases from which these corporations profit.
Advantages
 Viral gene therapy is capable of curing many diseases
 Providing a cost effective approach to curing Type I diabetes
  with minimal complications in comparison to a pancreas
  transplant. Using gene therapy abolishes the high risks of
  taking immunosuppressant drugs and would be assessable
  to a larger population pool. Currently, pancreas transplants
  are only available to individuals who are also eligible for a
  kidney transplant.
 Continuing to find cures for genetic diseases and possibly
  eliminating their existence
How it Contributes to Science
 Viral gene therapy helps us better understand viruses
 It can help us understand how viruses can be killed as well
  as how they infect certain cells
 If used in mass numbers, viral gene therapy has the potential
  of removing genetic disorders from the gene pool entirely
Conclusion
 Viral gene therapy, though it has its flaws, it can indeed lead
  to a better future for humanity
 To date, the therapeutic results of gene therapy have been
  minimal. This approach holds much potential, however,
  especially for treating single-gene disorders, and it is being
  actively investigated.
 I hope to one day see viral gene therapy replacing insulin
  pumps and pancreas transplants for type 1 diabetes.

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Viral gene therapy

  • 2. What It Is  Viral Gene therapy is the methodic use of viruses that normally cause illness as vectors to transfer genes that will produce desired effects in patients afflicted with certain illnesses.
  • 3. What It Is There is a lack of an efficient non-toxic delivery system to provide the successful application of gene therapy for treatment of a range of diseases has been a major barrier to gene therapy. Viruses have evolved to deliver their genes to target cells and are considered to be the most effective means of gene delivery and have an ability to be manipulated to express therapeutic genes or to replicate specifically in certain cells. Gene therapy is being developed for a range of diseases including inherited monogenic disorders and cardiovascular disease, but it is in the treatment of cancer that this approach has been most evident, resulting in the licensing of a gene therapy for the routine treatment of head and neck cancer in China.
  • 4. How It Works  Viral gene therapy works by first removing the genes of a viral vector then replacing it with genes that will produce the desired effect.
  • 5. What It Can Cure  Viral gene therapy is capable of curing a multitude of diseases, both genetic and acquired.  Diseases that viral gene therapy can cure include:  Cancer  Type 1 diabetes  Sever combined immune deficiency  Chronic granulomatous disorder  Hemophilia  Various cancers  Neurodegenerative diseases  Cystic fibrosis
  • 6. Viral Vectors Used  The two most commonly used vectors in the process are:  Retroviruses  Adenoviruses  Other less frequently used viruses are:  Adeno-associated viruses  Lentiviruses  Pox viruses  Alphaviruses  Herpes viruses  These viruses all differ in how well they are able to transfer genes to the cells they recognize and are able to infect, and whether they alter the cell’s DNA temporarily or permanently  These viruses are displayed on the following slide.
  • 7.
  • 8. Risks  There are a number of risk factors involved in viral gene therapy.  Such problems include:  Destruction of healthy cells in cancer treatment  Cancerous mutations due to injection at incorrect locations on DNA  Changes can be passed on to children if the reproductive cells are affected.  In addition, interference from corporations that produce drugs for people with chronic illnesses, such as Medtronics who produce insulin pumps. Viral gene therapy is not supported by the entities that fund research because it would be the cure to the very diseases from which these corporations profit.
  • 9. Advantages  Viral gene therapy is capable of curing many diseases  Providing a cost effective approach to curing Type I diabetes with minimal complications in comparison to a pancreas transplant. Using gene therapy abolishes the high risks of taking immunosuppressant drugs and would be assessable to a larger population pool. Currently, pancreas transplants are only available to individuals who are also eligible for a kidney transplant.  Continuing to find cures for genetic diseases and possibly eliminating their existence
  • 10. How it Contributes to Science  Viral gene therapy helps us better understand viruses  It can help us understand how viruses can be killed as well as how they infect certain cells  If used in mass numbers, viral gene therapy has the potential of removing genetic disorders from the gene pool entirely
  • 11. Conclusion  Viral gene therapy, though it has its flaws, it can indeed lead to a better future for humanity  To date, the therapeutic results of gene therapy have been minimal. This approach holds much potential, however, especially for treating single-gene disorders, and it is being actively investigated.  I hope to one day see viral gene therapy replacing insulin pumps and pancreas transplants for type 1 diabetes.