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

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

DNA Sequencing

DNA Sequencing Tutorial

Publishing the human genome

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

Success of Gene Therapy in Treating the Blind

YouTube: Gene Therapy

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