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IN THE NAME OF GOD Islamic Azad University of Falavarjan Department of Biology Biotechnology Dr. Keivan Beheshti Maal. Gene Therapy. Mahzad Tabesh November 2010. List of contents. Genetic disorder Gene therapy Types of GT Gene delivery systems Gene transfer vectors

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IN THE NAME OF GODIslamic Azad University of FalavarjanDepartment of BiologyBiotechnologyDr. Keivan Beheshti Maal
gene therapy
Gene Therapy

Mahzad Tabesh

November 2010

list of contents
List of contents
  • Genetic disorder
  • Gene therapy
  • Types of GT
  • Gene delivery systems
  • Gene transfer vectors
  • Genetic disorders treated with GT
  • Wound healing with GT
  • Immunology of GT
what is genetic disorder
What is genetic disorder?
  • Genes are made of DNA-the code of life.
  • Changes in sequence determine the inherited differences between each of us.
  • Some changes may cause serious disease like Cystic Fibrosis, Muscular Dystrophy and…
  • So genetic disorders are medical conditions caused by an error in a person’s genetic material.
what is gene therapy
What is gene therapy?
  • GT is the use of genes as medicine.
  • Thus GT can be defined as the use of genetic manipulation for treatment of diseases, instead of treating the symptoms of them.
how does gt work
How does GT work?
  • There are 3 main steps in each GT trial:

1. Isolate and clone the gene of interest.

2. Insert the gene into a vector to produce a “Chimera”.

3. Insert the Chimera into the patient’s body cells.

  • So functional proteins are created from therapeutic gene, causing the cell to return to a normal state.
approaches for correcting faulty genes in gt
Approaches for correcting faulty genes in GT
  • An abnormal gene could be swapped for a normal gene.
  • The abnormal gene could be repaired, returning the gene to it’s normal function.
  • The regulation (the degree to which a gene is expressed) of a particular gene could be altered.
types of gene therapy
Types of gene therapy
  • Somatic GT:

It will only change somatic cells in the body of the person being treated.

  • Germ line GT:

The therapeutic genes are transferred into the germ line cells (such as egg and sperms).

types of gene delivery systems
Types of gene delivery systems
  • In-vivo:

Genes will be delivered through a vector directly into the cells, while they are in the body.

  • Ex-vivo:

Delivery of genes to the cells will be out of the body.

vectors of gene therapy
Vectors of gene therapy
  • Viral vectors:
  • Retroviruses
  • Adenoviruses
  • Adeno-associated viruses
  • Herpes viruses…
  • Non-viral vectors:
  • Naked DNA
  • Liposomes
  • DNA-Gold particle bombardment (gene gun)…
examples of treated diseases with gt
Examples of treated diseases with GT
  • Severe Combined Immunodeficiency Disease (SCID)
  • Failure to develop functional T and B lymphocytes due to the absence of Adenosine Deaminase (ADA).
  • Accumulation of nucleotide metabolites= Toxic to developing T lymphocytes.
  • Death due to untreated severe infections.
  • GT aims at delivering ADA gene into the lymphocytes in the peripheral blood.
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Cystic Fibrosis
  • The most common congenital disease;the child’s lungs and pancreas and intestines become clogged with thick mucus.
  • Defective gene: Cystic Fibrosis Transmembrane Conductance Regulator gene.
  • CFTR makes a protein that controls the movement of salt and water in and out of the cells in the body.
  • GT aims at delivering CFTR gene directly into the epithelial cells lining airways in lungs.
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Familial Hypercholesterolemia (FH)
  • Patients have greatly elevated levels of serum cholesterol and suffer from heart attacks early in life.
  • FH is caused by absence or grossly malfunctioning low-density lipoprotein (LDL) receptors.
  • GT aims at delivering LDL receptor gene into the hepatocytes in the liver.
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Color Blindness
  • A condition in which certain colors cannot be distinguished.
  • Red-Green CB is the most common form(99%).
  • CB is the result of one or more of the cone cell color coding structures being absent or not functioning properly.
  • Usually CB is caused by absence of certain pigments (Opsin) in photoreceptor cells of Retina.
  • GT aims at delivering specific Opsin gene into the cone cells of the Retina in the eye.
better wound healing process with gt
Better wound healing process with GT

GT offers significant possibilities for the modification of adult wound healing processes by mimicking the cytokine profile of fetal wounds and by reducing the inflammatory responses to cutaneous injuries by shooting EGF and IGF genes into the wounds.

immunology of gene therapy
Immunology of gene therapy
  • Immunological Responses to GT includes both Humoral and Cell-mediated immunity.
  • These responses lead to many problems:
  • Transient expression of therapeutic gene
  • Non-efficient re-administration of the same vectors
  • Severe side-effects in clinical trials
immunology of gene therapy1
Immunology of gene therapy
  • Factors affecting the immune responses to gene transfer:

1.Host factors such as recipient immunogenotype, prior exposure to viruses, maturity of immune system and…

2.Gene transfer protocol factors such as route of transgenic delivery and type of delivery system.

3.Transgene delivery vehicle factors such as type of viral vector, presence of vector preparation contaminants, pseudo type of vector particle.