gene theraby is an approach to treat many diseases itis areal hope for millions of patients

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2. Gene
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A gene is the unit of hereditary
and carries inherited information

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Amazing Facts
3 billion (3,000,000,000) letters in the DNA code in every cell in your body.
There is 6 feet of DNA in each of our cells packed into a structure only 0.0004
inches across (it would easily fit on the head of a pin).
There are 100 trillion (100,000,000,000,000) cells in the body
If all the DNA in the human body was put end to end it would reach to the sun
and back over 600 times (100 trillion x 6 feet divided by 93 million miles = 1200).
If we recited the genome at one letter per second for 24 hours a day it would take
a century to recite the book of life.
The vast majority of DNA in the human genome - 97%- has no known function.

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5. Genes who are you Exactly?
I am carried on a chromosome
I am The basic unit of heredity
Encode how to make a protein
DNARNA proteins
Proteins carry out most of life’s function.
When altered causes dysfunction of a protein
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6. Chromosome to Gene
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7. Genes per chromosome
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8. Gene structure
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9. Gene structure
5‘UTR
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Protein coding sequence 3‘UTR
promotor exon 1 exon 2 exon n
exon n-1

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How Function is done

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12. Our life is maintained by molecular
network systems
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Molecular network
system in a cell

13. Proteins play key roles in a
living system
• Three examples of protein functions
• Catalysis:
Almost all chemical reactions in a
living cell are catalyzed by protein
enzymes.
• Transport:
Some proteins transports various
substances, such as oxygen, ions,
and so on.
• Information transfer:
For example, hormones.
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Alcohol
dehydrogense
oxidizes alcohols
to aldehydes or
ketones
Hemoglobin
carries oxygen
Insulin controls
the amount of
sugar in the
blood

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TERTIARY STRUCTURE (fold)
TERTIARY STRUCTURE (fold)
Genome
Expressome
Proteome
Metabolite
Functional Genomics
From gene to function

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16. Dilemma
• When there is a mutation in the gene, then it will
change the codon, which will change which amino
acid is called for which will change the conformation
of the protein which will change the function of the
protein. Genetic disorders result from mutations in
the genome.
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17. Imagine
• Imagine that you accidentally broke one of
your neighbor's windows.
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18. let us imagine with each other
What will you do?
Stay silent: no one will ever find out that you
are guilty, but the window doesn't get fixed.
Repair it with some tape: not the best long-term
solution.
Put in a new window: not only do you solve the
problem, but also you do the honorable thing.
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19. What is similar in gene therapy
I. Stay silent: ignore the genetic disorder
and nothing gets fixed.
II. Try to treat the disorder with drugs or
other approaches: depending on the
disorder, treatment may or may not be a
good long-term solution.
III. Put in a normal, functioning copy of the
gene: if you can do this, it may solve the
problem!
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20. How to fix it
A
B C A a beneficial gene
virus modified virus
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21. A virus is found which replicates by inserting its genes into the host cell's genome. This virus
has three genes - A, B and C.
Gene A encodes a protein which allows this virus to insert itself
into the host's genome.
Genes B and C actually cause the disease this virus is associated with.
Replace B and C with a beneficial gene. Thus, the modified virus could introduce your 'good
gene' into the host cell's genome without causing any disease.
So we use the modified virus to fix the “broken window”
Gene therapy using an Adenovirus
vector.
A new gene is inserted into an
adenovirus vector, which is used to
introduce the modified DNA into a
human cell. If the treatment is
successful, the new gene will make
a functional protein.
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22. Gene Therapy
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23. Diseases
Which of the following diseases are current or potential targets of
gene therapy?
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Acquired
Inherited diseases
a. Cancer b. AIDS c. Diabetes d. Parkinson’s
Disease

24. Ex
1-one gene
Cystic fibrosis
Severe combined immunodeficiency
Adenosine deaminase and purine nucleoside
phosphorylase
2- gene therapy for cancer
3-gene therapy for cardiovascular diseases
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25. Diseases
• Acquired Inherited
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26. Diseases
Genetic diseases:
Type 1: Single locus (gene) is defective and responsible for
the disease, 100% heritable.
: Sickle cell anemia,
Hypercholesterolemia
Cystic fibrosis
Type 2: Polygenic traits, <100% heritable, may be dependent
on environmental factors and lifestyle.
Heart disease
Cancer
Diabetes
Alcoholism
Schizophrenia
Criminal behavior
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27. QUESTIONS
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28. What is Gene Therapy formal
definition
It is a technique for correcting defective genes
that are responsible for disease development
There are several approaches:
1-A normal gene inserted to compensate for a
nonfunctional gene.
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29. techniques
-An abnormal gene
changed with normal gene
An abnormal gene repaired through
selective reverse mutation
-Change the regulation of gene pairs
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30. • Insertion of genes into individual’s cell
• Or
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Tissue
To treat a disease
Hereditary disease in particular

31. Gene Therapy mainly aim to
• Supplement a defective mutant allele
• With a functional one
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32. • diseases treated by gene thereby
• Can be divided to
• inherited Acquired
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Diseases like
parkinsonism
AIDS
Diabetes