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Human Genome Project and Germ Line Therapy

Prepared by: LJ Silvestre

Genes and the HGP


Deoxyribonucleic Acid (DNA) comprises of four distinct compounds called bases, which are denoted: A, T, G and C. Genes, which store the information on the building of proteins can be located on certain DNA stretches. The entire DNA, which includes the genes in an organism, makes up its genome. So the human genome is a collection of all human genes, consisting of DNA, contained within the 23 chromosome pairs. Genes are often described as the biological blueprints or recipes for life and are found in the DNA, carrying the genetic information from one generation to the next. Many people are convinced that genes are special, that they contain a person's essence, which has enormous spiritual and commercial value. We are who we are because of our genes.

Genes and the HGP


With advances in molecular genetics, it became possible to launch the Human Genome Project (HGP) - a sequencing project that determines the genetic makeup of an organism by reading off the sequence of the three billion DNA bases, which encode all of the information necessary for the life of the organism. The sequence of our genome will ultimately allow us to discover the secrets of life's processes, the biochemical basis of our senses and our memory, our development and our aging, our similarities and individual differences.

Genes and the HGP


The genome project itself offers no promises of cancer cures or quick fixes for Alzheimer's disease, and no detailed understanding of genius or schizophrenia. But this is the start to possibly prolonging our lives and even creating synthetic life.

Germ Line Gene Therapy


Gene therapy is an experimental technique used in treatment of malfunctioning genes, whereby a deficient or defective gene is replaced by a working gene, so that the body can make the proper enzyme or protein and as a result eliminate the origin of the disease. In germ line gene therapy, Germ cells, i.e., sperm or eggs are modified by the introduction of functional genes, which are integrated into their genomes. This would allow the therapy to be heritable and passed on to later generations. Although this should, in theory, be highly effective in counteracting genetic disorders and hereditary diseases Implies custom-made children.

Gene Therapy Procedure


The technology of gene therapy is based on the effective delivery of the corrective genes and to do this, scientists have developed gene delivery vehicles called vectors. These vectors encapsulate therapeutic genes for delivery into the target cells. Many of the vectors currently in use are based on attenuated or modified versions of viruses. Plasmids, which are circular pieces of DNA extracted from bacteria, are also used as vectors.

The therapeutic gene to be transferred is extracted from the cell of a healthy individual. The gene is extracted by cutting the DNA using a restriction enzyme (restriction enzymes "digest" DNA at designated nucleotide locations along the DNA chain). There are different types of restriction enzymes, each being specific to the location of the DNA chain that it will cut. The section of cut DNA has to be intact then a technique called electrophoresis is used to separate the selected pieces of DNA and remove the genes that contained the DNA sequence that coded for the polypeptides needed.

Similar restriction enzymes are used to remove a section of enzyme from a plasmid or virus and using ligase (a special enzyme used to "glue" a foreign piece of DNA into a donor cell) the therapeutic DNA sequence is placed in the DNA of the vector. In the case of a virus, instead of causing illness the virus carries the normal genes into the target cells where they begin functioning.

Ex Vivo
There are methods of inserting genes, such as addition of chemicals to allow the large DNA molecules in solution to pass through the cell membrane. This requires that the cells be removed from the body and treated in a laboratory, making it suitable for treatment of easily transplantable tissues such as blood and bone marrow. This type of gene therapy is referred to as ex vivo, as the cell to be implanted are harvested from the patient, modified and then replaced.

In Vivo
A technique that involves direct insertion of the vector into the patient is referred to as in vivo gene therapy. There are also other techniques, which include laser micropuncture of the cell membrane, electroporation and biolistics (using high velocity tungsten microprojectiles to insert DNA)

Some Ethical Concerns


To treat a pre-embryo that carries a serious genetic defect before implantation into the mother (this requires the use of in vitro fertilization techniques) To treat the germ cells (sperm or egg cells) of the afflicted adults so that their genetic defects would not be passed on to their offspring. This approach requires the technical expertise to remove the defective genes and insert a properly functioning replacement.

Activity
Pair up On a sheet of paper, list down at least 3 ethical concerns connected with HGP and the Germ line therapy

Questions
We can now identify who has what type of sickness, what kind of ethical concerns (in finding a job, family, etc.), are connected to this? And given these concerns should we still pursue the HGP?

Questions
There is a way for us to live without any sickness, prolong our lives and manipulate the way our children will come out. Again, what sort of issues may arise and given those issues, should we still pursue HGP and Gene Therapy?

References
http://www.ndsu.edu/pubweb/~mcclean/plsc431/students99/rothe.htm

http://sgugenetics.pbworks.com/w/page/38231663/The%20Human%20Genome%20Project
http://www.montegen.com/Montegen/Nature_of_Business/The_Library/Genomics/Germ_Li ne_Gene_Therapy/germ_line_gene_therapy.htm

http://www.angelfire.com/hi3/germline/ http://www.youtube.com/watch?v=XuUpnAz5y1g&list=LL1S6I6KafLSwEVE2lkHjYeA&index=1 &feature=plpp_video (Human Genome Project)

http://www.youtube.com/watch?v=N4i6lYfYQzY&list=LL1S6I6KafLSwEVE2lkHjYeA&index=1&f eature=plpp_video (How to Sequence a Genome: Introduction)

http://www.youtube.com/watch?v=EfXK50Bxod8&feature=related (Gene Therapy Example) http://www.youtube.com/watch?v=Ce8ZVyUqY-I&feature=relmfu (Craig Venter)

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