Gene Therapy

Contents
• What is meant by Gene Therapy
• How does Gene Therapy work
• How does Gene Therapy Work
• Types of Gene Therapy
• Kinds of Gene Augmentation
• Shortcoming of Gene Therapy
• Ethical Debate regarding Gene Therapy
• Bibliography
What is meant by Gene Therapy?
• Simply Put: Gene Therapy is the therapy for the genes by the genes.
• ??? !!!! ????
• This is the new field which is also known as Biotech or
Biopharmaceuticals.
• It involves in the manufacture of Proteins which:
• Provide a DNA strand / gene which provides a cure for a disease
• Replaces the damaged or malfunctioning gene in the designated cell DNA
• Activates or deactivates the required gene in a cell
• Causes the body to attack a certain type of cell by changing the operation of
that type of cell.
How does Gene Therapy work
• So…. it is simply using proteins to cure a disease. And all meat is
protein so if we eat meat we are cured of all diseases?
• RIGHT??
• Wrong. While meat is protein, that is neither the kind of protein
required nor that is the method of administration.
• The proteins are very sensitive to pH and are very easily denatured by
the saliva in the mouth or the acid in the stomach.
• The method of administration of proteins is either intravenous or by
direct injection at the required site. But that is just half the story.
How does Gene Therapy work
• The protein must by carried by a virus (a
retrovirus or adenovirus) through the cell
into the nucleus for the gene to work.
• If the protein is just injected into the cell, it
does not work.
• In Gene Therapy specialized proteins either
from animal, human or plant sources are
used or synthetic proteins are developed in
the laboratory under special conditions.
Type of Gene Therapy
• Gene Augmentation Therapy
• In this type of therapy the required gene is made part of the cell DNA.
• It can either replace the existing cell DNA
• It can either add additional DNA to the existing DNA
• Gene Inhibition Therapy
• In this type of therapy the either:
• The faulty gene is deactivated
• The product of the faulty gene is counter acted by the protein.
• Cell Killing Therapy
• In this therapy, the protein acts either:
• Becoming toxic as soon as it enters the required cell and kills it.
• Makes the required cell a target for the body immune system and has it killed.
Kinds of Gene Augmentation
• There are two kinds of gene Augmentation:
• Somatic Gene Therapy
• This is where the modified cells are not part of the reproductive system or stem cells and
are not passed down from parents to kids
• Germline Gene Therapy
• This is where the modified cells effect the sperms, eggs or the stem cells and the
modifications are passed on to the next generations
Shortcoming of Gene Therapy
• It doesn’t always work because:
• All humans are different and while it works in some, it causes adverse effects
in others causing:
• Toxicity including death
• Inverse reaction to the intended treatment
• Killing of the wrong cells
• Activation of cancerous growths
• The DNA is very complex and has not been fully decoded as yet. In
some cases the required gene does not find the DNA to replace.
• Mutations in every human being, so everyone does not have the
same kind of genes.
Ethical debate regarding Gene Therapy
• Should it be allowed?
• To what extent should we use it?
• Who decides what is ethical and what is not?
• Who decides what is acceptable modification?
• What is medicine and what is warfare?
Bibliography
• Genetics Home Reference - https://ghr.nlm.nih.gov/
• http://www.yourgenome.org/facts/what-is-gene-therapy
THEEND