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Human Genome Project, Gene Therapy, and Cloning Spring 2009 Adapted from the University of Utah Genetic Science Learning Center and The National Genome Research Institute, National Institutes of Health The Human Genome Project An international effort to decipher the DNA blueprint of a human being 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 Publishing the human genome 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. 3. The virus uses the normal sequence to produce the missing protein and the cell returns to normal. Boy in the Bubble SCID – Severe Combined Immune Deficiency 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 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 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 (46 chromosomes) by electric shock 3. The new fused cell begins dividing 4. The embryo is placed in the uterus of a foster mother and allowed to develop normally 5. The foster mother gives birth to a genetic clone of the original donor!