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
Spring 2010
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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DNA Sequencing
DNA Sequencing Tutorial
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Publishing the human genome
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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 (remember cystic fibrosis).
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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Success of Gene Therapy in Treating the Blind
YouTube: Gene Therapy
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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
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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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