Transcript Human Genome Project, Gene Therapy, and Cloning

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?

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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!