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Human Genome Project,
Gene Therapy, and Cloning
Adapted from the University of Utah Genetic Science Learning Center
and The National Genome Research Institute, National Institutes of Health
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The Human Genome Project
• An international effort to decipher the DNA
blueprint of a human being
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Human Genome Project Goals:
• To sequence & determine the exact order of the
nucleotides (A,C,T,G) for ALL of the DNA in a human
cell
• To determine which sections of DNA represent the
individual genes
• To store this information in databases for analysis
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Publishing the human genome
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How genes influence an
individual’s response to
drugs
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Gene
Therapy
Replacement of an
abnormal protein with
a normal one
1. Isolate the normal sequence for a gene and package it
into a virus (vector).
2. Infect a target cell, usually the one with the illness, such
as a liver or lung cell.
3. The cell uses the normal sequence to produce the
missing protein and is thus repaired.
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Problems with Gene Therapy
• Short lived, so patients have to go through multiple
rounds
• Immune response to foreign invaders
• Problems with the viruses
• Very expensive
• Multigene disorders: disorders like heart disease, high
blood pressure, Alzheimer’s disease, diabetes are
caused by the combined effects of variations in many
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diseases.
What is Cloning?
• Cloning is the creation of an organism that
is the EXACT genetic copy of the mother
• Identical twins are natural clones, they
share 100% of their DNA
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How do you clone an organism?
1. Remove the nucleus from an egg cell and a
regular cell from a donor.
2. Fuse the enucleated cell with a regular cell (that
still has its nucleus) by electric shock
3. The new fused cell begins dividing, just as a
fertilized egg would.
4. The embryo is placed in the uterus of a surrogate
mother and allowed to develop normally.
5. The surrogate mother gives birth to a genetic
clone of the original egg cell donor donor!
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