Human Genome Project, Gene Therapy, and Cloning
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Transcript of Human Genome Project, Gene Therapy, and Cloning
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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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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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Gene Therapy
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.
Replacement of an abnormal protein with a normal one
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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 diseases.
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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!