Transcript Human Genome Project, Gene Therapy, and Cloning

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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