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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

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!

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