Genetic Engineering for Treatment or Cure of Genetic Diseases

Lawrence Chillrud and Skylar Karzhevsky Period 9

Basic Principles of Genetics1. Traits that offspring inherit are controlled by two types of alleles: Dominant,

and Recessive alleles. Alleles are different forms of a gene. When you cross two parents their genetic material (DNA) is crossed, which means that their alleles are crossed. The alleles are either dominant or recessive. When Dominant alleles show up in the cross they “mask” or “overpower” the recessive alleles. For example if purple hair- the dominant allele is crossed with pink hair- the recessive allele, then the purple hair will show up in the offspring. If both parents have pink hair- the recessive allele, then the recessive allele will show up in the offspring. So in conclusion, if there is even one dominant allele in the cross then the dominant allele will show up, but if there are two recessive alleles in the cross then the offspring will inherit the recessive allele.

2. Co-dominant alleles are different than dominant and recessive alleles. When two co-dominant alleles are crossed they don’t “mask” or “overpower” each other. Instead they are equally strong and both show up in the offspring. For example- if you have a white chicken crossed with a black chicken, neither of the alleles are dominant or recessive. Instead of the offspring having white or black feathers it will have both white and black feathers.

Basic Principles of Genetics (ctd.)3. Two alleles containing certain traits, one from each parent

combine to pass a trait on to their offspring. Each allele that contains the trait is either dominant or recessive. Whether the alleles are dominant or recessive will determine which traits the offspring will have. Dominant alleles mask recessive alleles whenever they are present. If the offspring receives two dominant alleles from its parents, the offspring will have whichever trait those alleles contain. If the offspring has two of the same alleles for one trait, it is homozygous. If the offspring has one dominant allele, and one recessive allele, the offspring will have the dominant trait because it will mask the recessive trait. If the offspring has different alleles, it is heterozygous. If the offspring has two recessive alleles, the offspring will have the recessive trait, and will also be homozygous.

Human Genome Project1. The Human Genome project began in October of 1990. When the project

originally began, the scientists’ goals were to sequence, or decode the human genome. Scientists wanted to identify the 20,000-25,000 genes that are contained in human DNA and find the sequences of the three billion chemical base pairs that make up human DNA.

2.      Some implications in regards to legal, ethical, and social implications are that companies didn’t know or questioned: the fairness in the use of genetic information, privacy and confidentiality of genetic information, psychological impact the project has on the person, reproductive issues, clinical issues uncertainties concerning tests, health and environmental issues, and commercialization of products such as copyrights etc.

3. The Human Genome Project changed privacy laws such as GINA when they finally decoded the human genome. Because of the Human Genome Project insurance companies were now able know if a person carried a certain gene for a disease. Since the insurance company knows The Genetic Information Discrimination Act prevents this from happening.

Genetic Disorder1. 1.      single gene disorder- A disease caused by abnormality in one

gene. Ex. Cystic fibrosis2.    chromosome abnormalities- typically occur when a chromosome is missing or there is an extra chromosome. Ex. Down’s syndrome3.    multifactorial disorders- mutations in multiple genes, which may interact with environmental factors. Tend occur later in life. Ex. Alzheimer's

2. If parents with genetic disorders want to know the chance of their children getting the same disease, they can get genetic testing that can tell them about any abnormal chromosomes, or whether they carry a gene for a disorder. Genetic counselors could also analyze the pedigree of both parents, to determine the probability of heredity for genetic disorders or problems. If they do test positive for any genetic abnormalities, they can go to genetic counseling to determine whether or not or how they should have children. If the probability for having a child with any genetic disorders is high, the couple will most likely not have a child naturally.

Genetic Disorders (ctd.)

3. A karyotype is the characterization of the chromosomes of a species. After a karyotype has been made in a fetus, amniocentesis can be used to test the amniotic fluid surrounding a fetus for abnormal karyotypes that lead to genetic disorders such as Down’s syndrome, Kleinfelter’s Syndrome, and Turner’s syndrome.

Argument- Pro Gene Therapy

Basically the whole point of gene therapy is to cure genetic disorders such as Parkinson's disease, Alzheimer's disease, and many more. There are thousands of diseases in this world, and we are constantly trying to cure them. We have cures for some, and are still trying to find cures for others. But we can’t cure genetic disorders with medicine, or drugs or any of that stuff. The only way to cure genetic disorders is to replace the defective gene with the correct gene. That’s why scientists are studying gene therapy. Mark Kay, the director of a human gene therapy program at Stamford University School of Medicine and one of the founders of the American Society of Gene and Cell Therapy has a pretty positive attitude about gene therapy; “There is a lot of optimism in the gene therapy field right now that these therapies will work well.” “There are a lot of conditions for which gene therapy could work. The easiest target appears to be single gene disorders, including adrenoleukodystrophy and blindness, where only a small percentage of cells need to be fixed.” There have been multiple studies on gene therapy and in one scientific experiment; scientists have been able to let monkeys Sam and Dalton the ability to see the colors red and green.  “The achievement is technically amazing and conceptually very cool,” says Melissa Saenz, a neuroscientist at Caltech in Pasadena, California. “Although color blindness is a relatively mild defect, this type of therapy may shed some light on treating more severe vision disorders,” says Jay Neitz, a visual neuroscientist from the University of Washington. Gene therapy can potentially wipe out a disease before it can begin and prevent future generations from getting the disease. So as you can see gene therapy can be very effective.

Argument- Pro Stem Cell Therapy

Stem Cell therapy is basically where cells are taken from you, altered a little, and put into damaged parts of your body to grow and multiply, it’s kind of like regenerating your body parts. There are many pros to stem cells, for example, it as the potential to treat a number of medical conditions, it also teaches scientists about the growth of cells and their development. Stem cells might eliminate test subjects for medicine products, the drugs and such products can be tested on the stem cells instead of a rat or human. Also since the original cells are being taken from the patients own body there is a lower chance of the cells being rejected when put back in the body. One study that scientists from the Georgetown University Medical Center have been working on is to create retinal cells out of stem cells. Nady Golestaneh, an assistant professor in the department of biochemistry and molecular and cellular biology at Georgetown pretty much sums up the answer: “We have shown that we are able to generate retinal cells from cells originally taken from a small amount of biopsied skin, that are then induced to become stem cells,” the surprising thing is the cells actually work “The retinal cells we have generated are really functional,” says Golestaneh. The future looks bright for stem cells.

Argument- Pros of Organ Cloning

Organ cloning is just what it sounds like- Organ cloning! Basically you take some of your own organ, clone the cell and make an exact copy of it. There is almost no chance of the organ being rejected because the organ is you; it has the same DNA so your body won’t think of it as an enemy. If you clone yourself then you don’t need a donor, and with no donor means no second surgery. There is also no waiting in line for a transplant; you are the only one in line. Statistics on the website http://www.organdonor.gov/ show that 110,460 people are waiting for an organ and 18 people die every day waiting for one. Luckily due to pigs, those people might not have to wait that long. Pigs are special because with a little bit of tweaking we can modify the pigs chromosomes, and make it where we can grow our organs in the pigs, and then transplant the organ from the pigs into us. Several organizations have said they have successfully cloned pigs. So as cloning advances, so does peoples chances of surviving.

Argument 4 Pro

Argument Against Genetic Engineering- It’s Not Natural, and It May Be Unsafe

Dr. John Fagan, a professor of Molecular Biology at Maharishi University of Management says “When genetic engineers disregard the reproductive boundaries set in place by natural law, they run the risk of destroying our genetic encyclopedia.” One of the dangers of genetic engineering is changing the way of nature. Nature creates things for a purpose, and changing it may bring some unexpected side effects to procedures such as cloning, or trying to destroy genes that carry certain diseases. Scientists must be careful- by attempting to cure a disease, they may be accidentally helping another, more dangerous disease to cause harm to the person. Also, one might never know what could happen. Genetic engineering procedures have not yet been perfected. The dangers of genetic engineering are extensive. Messing with Nature’s laws could bring serious consequences, because most things happen for a reason, and scientists can never fully understand the way that nature works.

Argument- The cons of Gene Therapy

Though gene therapy could save many lives, it could also backfire and end a life as well. Gene therapy is the process of eliminating, or introducing a new gene into the human body. If person were to have a certain disease, a new gene could be introduced into their body to try and prevent this disease from becoming stronger. However, it has been proven that this new gene could also help a second, unknown disease to become stronger and potentially harm the person. In addition to backfiring, many people are against gene therapy because in using it, scientists could make “designer babies” by inserting genes into a fetus. Parents are able to choose which traits that they would like their children to have. For instance, athletic ability, intelligence, perhaps even blonde hair and blue eyes. Many scientists, such as Dr. Zev Rosenwaks, a director of the Center of Reproductive Medicine and Infertility at Cornell medical center in New York believe that gene therapy should be used for medical purposes only. "Our view at the moment is that these techniques should be used for medical indications, not family balancing.“ says Dr. Rosenwaks. Many people believe that in making “designer babies”, people will lose their individuality. Gene therapy may be used to save lives, but it could also potentially end them, and help the people in our world create babies according to what they want.

This diagram explains how the process of Gene Therapy works

Argument- The cons of Therapeutic Cloning, also known as Stem Cell transplants

A scientist extracting the nucleus from a somatic cell

In therapeutic cloning, scientists extract the nucleus from an egg cell from either an animal or a human. Scientists then take a somatic cell (non sex cell, body cell) from the patient and extract the nucleus from it as well. The nucleus removed from the patient is then inserted into the egg cell from the donor. The egg now contains the genetic material from the patient, and is stimulated to divide, and create several new cells that are infused into the patient. It often could take perhaps dozens of times to get the egg to grow and thrive. One of the problems with therapeutic cloning is that in extracting the nucleus from the egg cell, an embryo is being killed. This is an object of controversy especially among religious groups, and have compared the destruction of the embryo to an abortion, because the embryo could potentially become a human being. Another problem is, that in learning how to clone cells, in the future, scientists could easily learn how to clone a human being. Also, in diseases such as Alzheimer’s in which cells are dying or being disrupted, the transplanted cells may do the same, and the procedure could be pointless. Professor Harvey, a former chair of the WA Reproductive Technology council says “In the case of diseases such as Alzheimer’s, unless we understand why cells are dying and circuits are becoming disrupted, any transplanted cells may be subject to the same degenerative processes. Some neurological conditions may have an auto-immune component and, if there is, cloned cells may turn out to be the wrong cells to transplant unless previously manipulated in some way. Transplantation really is a last resort. In my view, earlier diagnosis and prevention of further degeneration is the optimal strategy.” Therapeutic cloning does not usually cause harm to the patient, it may not always work the way the scientists wish it to.

As shown in the chart, not one hundred percent of all stem cell transplants are effective. In fact, 14 out of 50, or 28% of these Parkinson’s patients did not experience any change.

Argument- The cons of using pig organs for organ transplants

Though using genetically modified pig organs for humans can be useful, there is one danger. According to Dr. Daniel Salomon of the Scripps Institute reported that “Certain viruses unique to pigs can infect human cells. While scientific technology may make it possible to transplant human organs into humans, significant safety concerns still exist.” Some viruses found in pigs, such as PERV (pig endogenous retroviruses) are found in pig cells, and can be easily passed on to humans through the transplant of organs such as kidneys, livers, and lungs. There are approximately 50 PERV per pig genome, so there is a good chance that the human patient could be infected with PERV. It is completely possible for the virus to be removed from the pig’s genome, but it is a daunting and tedious task to perform, and is not usually done because of the high number of viruses in the genome. PERV is also very contagious, and can be easily spread through coughing or sneezing. A human infected with the virus could infect the rest of the community. Scientists are worried about diseases such as PERV being transmitted from pigs to humans becoming worldwide epidemics. There is no way to screen the pig organs or the human patients for unknown viruses. They could possibly remain dormant for months, or even years. Using pig organs may save lives, but many believe that the risks are too high for this procedure to be used.

The PERV Virus

Pig hearts look very similar to human hearts, and they are about the same size.

Scientific Advancements in Cloning

Since the famous cloning of Dolly the sheep in 1996, there have been many more scientific advancements since then. For example, organ cloning is now an option for people in need of an organ transplant. Scientists are able to remove a bit of tissue from the organ that they need removed, and from the cells in the tissue, clone the organ. This is much more reliable (though much more expensive) than finding an organ donor, because sometimes, the patient’s immune system will reject the organ. The body will stop functioning, and will soon die. Because of organ cloning, people do not have to wait a long time on organ donation waiting lists. Cloning is also used in the agricultural field. Plants are sometimes genetically modified to become riper faster, grow larger, having a longer shelf life, or taste better. Cloning allows these genetically enhanced plants to be mass produced, making a profit for many food production companies. Scientists have also found ways to clone domestic animals such as dogs for families who want a pet similar to the one that they currently own. Dogs are one of the most complicated animals to clone, because of their complex reproductive systems. Scientists figure that if dogs can be cloned, then almost any kind of cloning is possible.

Controversy About Genetic Engineering

There has been much controversy related to genetic engineering, but perhaps the most common is about human cloning and “designer babies”. The main reason that people are opposed to cloning humans and hand-picking the traits of children, is because that in doing this, the people of the human race will lose their individuality. Controversy related to religious matters is also common in genetic procedures such as stem cell transplants. When the nucleus is extracted from the egg, the cell dies. The cell is an embryo, and could potentially grow into a human being. This is viewed by many religious groups as murder, and is considered to be abortion. Agricultural engineering has also made waves throughout environmental organizations. Engineering plants to become riper more quickly, larger, or even to have a longer shelf life could potentially cause harm to humans. Also, genetically engineered plants require more chemicals to grow, which could harm the eco-system around them, causing many environmental organizations such as the EPA (Environmental Protection Agency) to take action.

Conclusion

Federal funding should be available for genetic engineering. If it is provided, the future scientific innovations and discoveries could be endless. Waiting lists for organ donors would be reduced immensely, and people could always be able to have an organ ready to transplant if need be. Though federal funding would be an open door for scientists to use genetic engineering for their own personal gain, there are already laws that prohibit those behaviors. Think about how much better the world could be if geneticists found a way to cure HIV/AIDS or cancer with gene therapy or stem cell transplants! Genetic Engineering has the potential to save millions of lives, so federal funding should be provided for this brilliant and innovative branch of science.

Works Cited 1• Cooper, M. H. (2000, May 12). Human genome research. CQ Researcher, 10, 401-424. Retrieved

from http://library.cqpress.com/cqresearcher/• Feero, Greg. "A perfect bookend." Skin & Allergy News 39.9 (Sept 2008): 16(1). General

OneFile. Gale. Tappan Zee High School Library. 13 Mar. 2011 http://find.galegroup.com/gps/start.do?prodId=IPS&userGroupName=nysl_se_tap

• "About the Human Genome Project." Oak Ridge National Laboratory. 19 Aug. 2008. Web. 20 Mar. 2011. <http://www.ornl.gov/sci/techresources/Human_Genome/project/about.shtml>.

• "What Do Scientists Say about the Dangers of Genetic Engineering?" Dangers of Genetic Engineering—Campaign for Mandatory Labeling of Genetically Engineered Food. Web. 24 Mar. 2011. <http://www.safe-food.org/-issue/scientists.html>. Primary Source

• "Engineering Humans." SFSU WWW Home Page Is Not on This Server. Web. 24 Mar. 2011. <http://online.sfsu.edu/~rone/GEessays/engineerhumans.html>.

• Highfield, Roger. "Dolly Creator Prof Ian Wilmut Shuns Cloning - Telegraph." Telegraph.co.uk - Telegraph Online, Daily Telegraph and Sunday Telegraph - Telegraph. Web. 24 Mar. 2011. <http://www.telegraph.co.uk/science/science-news/3314696/Dolly-creator-Prof-Ian-Wilmut-shuns-cloning.html>.

• "Monkeys Cured of Color Blindness." Current World News. Web. 24 Mar. 2011. <http://420chan.blogspot.com/2010/11/monkeys-cured-of-color-blindness.html>.

• "Q&A: Gene Therapy Turnaround - The Scientist - Magazine of the Life Sciences." The Scientist : Home : Thursday. Web. 24 Mar. 2011. <http://www.the-scientist.com/blog/display/56145/>.

• "Colorblind Monkeys See New Light - The Scientist - Magazine of the Life Sciences." The Scientist : Home : Thursday. Web. 24 Mar. 2011. <http://www.the-scientist.com/blog/display/55981/>.

Works Cited 2• "Cloning Fact Sheet." Oak Ridge National Laboratory. Web. 24 Mar. 2011.

<http://www.ornl.gov/sci/techresources/Human_Genome/elsi/cloning.shtml#organsQ>.• "Researchers Clone Pigs - CNN." Featured Articles from CNN. 16 Aug. 2000. Web. 24 Mar.

2011. <http://articles.cnn.com/2000-08-16/health/pig.clones_1_clone-pigs-pig-hearts-pig-organs?_s=PM:HEALTH>.

• "Scientist Solutions - Germline Gene Therapy." Scientist Solutions. Web. 24 Mar. 2011. <http://www.scientistsolutions.com/t11892-germline gene therapy_.html>.

• "Therapeutic Cloning - Explore Stem Cells (UK)." Understanding Stem Cell Research and Treatments at Explore Stem Cells (UK). Web. 24 Mar. 2011. <http://www.explorestemcells.co.uk/TherapeuticCloning.html>.

• "60% PARKINSON’S PATIENTS IMPROVE AFTER REPAIR STEM CELL TREATMENT – PHYSICIANS CONFIRM RESULTS! « The Stem Cell Blog." The Stem Cell Blog. Web. 24 Mar. 2011. <http://repairstemcell.wordpress.com/2010/02/06/60-parkinsons-patients-improve-after-repair-stem-cell-treatment-physicians-confirm-results/>. (Primary Source!)

• "Pros and Cons of Stem Cell Therapy." Health Guidance - Free Health Articles. Web. 24 Mar. 2011. <http://www.healthguidance.org/entry/12366/1/Pros-and-Cons-of-Stem-Cell-Therapy.html>.

• 24, Posted: March. "Progress Seen in Creating Eye Cells From Stem Cells - US News and World Report." Health News Articles - US News Health. Web. 24 Mar. 2011. <http://health.usnews.com/health-news/managing-your-healthcare/womens-health/articles/2011/03/24/progress-seen-in-creating-eye-cells-from-stem-cells?PageNr=1>.

Works Cited 3• "Gene Therapy." Oak Ridge National Laboratory. Web. 24 Mar. 2011.

<http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml>.• "Of Pigs, Primates, and Plagues: Xenotransplantation Critique." Medical Research

Modernization Committee. Web. 24 Mar. 2011. <http://www.mrmcmed.org/pigs.html>.• "Infection of Nonhuman Primate Cells by Pig Endogenous Retrovirus." Pubmed.gov. Web. 24

Mar. 2011. <http://www.ncbi.nlm.nih.gov/pubmed/10906227>.• "How Many People Die Each Year Waiting for Kidney Transplants? - Kidney Health Transplant

| Ask MetaFilter." Ask MetaFilter | Community Weblog. Web. 24 Mar. 2011. <http://ask.metafilter.com/70090/How-many-people-die-each-year-waiting-for-kidney-transplants>.

• Welcome to Organdonor.gov | Register Today as an Organ Donor. Web. 24 Mar. 2011. <http://www.organdonor.gov/>.

• "Pros and Cons of Stem Cell Therapy." Health Guidance - Free Health Articles. Web. 25 Mar. 2011. <http://www.healthguidance.org/entry/12366/1/Pros-and-Cons-of-Stem-Cell-Therapy.html>.

Works Cited 4• "Cloning Fact Sheet." Oak Ridge National Laboratory. Web. 24 Mar. 2011.

<http://www.ornl.gov/sci/techresources/Human_Genome/elsi/cloning.shtml#organsQ>.• "Researchers Clone Pigs - CNN." Featured Articles from CNN. 16 Aug. 2000. Web. 24 Mar.

2011. <http://articles.cnn.com/2000-08-16/health/pig.clones_1_clone-pigs-pig-hearts-pig-organs?_s=PM:HEALTH>.

• "Scientist Solutions - Germline Gene Therapy." Scientist Solutions. Web. 24 Mar. 2011. <http://www.scientistsolutions.com/t11892-germline gene therapy_.html>.

• "Therapeutic Cloning - Explore Stem Cells (UK)." Understanding Stem Cell Research and Treatments at Explore Stem Cells (UK). Web. 24 Mar. 2011. <http://www.explorestemcells.co.uk/TherapeuticCloning.html>.

• "60% PARKINSON’S PATIENTS IMPROVE AFTER REPAIR STEM CELL TREATMENT – PHYSICIANS CONFIRM RESULTS! « The Stem Cell Blog." The Stem Cell Blog. Web. 24 Mar. 2011. <http://repairstemcell.wordpress.com/2010/02/06/60-parkinsons-patients-improve-after-repair-stem-cell-treatment-physicians-confirm-results/>.

• "Pros and Cons of Stem Cell Therapy." Health Guidance - Free Health Articles. Web. 24 Mar. 2011. <http://www.healthguidance.org/entry/12366/1/Pros-and-Cons-of-Stem-Cell-Therapy.html>.

• 24, Posted: March. "Progress Seen in Creating Eye Cells From Stem Cells - US News and World Report." Health News Articles - US News Health. Web. 24 Mar. 2011. <http://health.usnews.com/health-news/managing-your-healthcare/womens-health/articles/2011/03/24/progress-seen-in-creating-eye-cells-from-stem-cells?PageNr=1>.

Works Cited 5• "Researchers Clone Pigs - CNN." Featured Articles from CNN. 16 Aug. 2000. Web. 24 Mar.

2011. <http://articles.cnn.com/2000-08-16/health/pig.clones_1_clone-pigs-pig-hearts-pig-organs?_s=PM:HEALTH>.

• "Scientist Solutions - Germline Gene Therapy." Scientist Solutions. Web. 24 Mar. 2011. <http://www.scientistsolutions.com/t11892-germline gene therapy_.html>.

• "Therapeutic Cloning - Explore Stem Cells (UK)." Understanding Stem Cell Research and Treatments at Explore Stem Cells (UK). Web. 24 Mar. 2011. <http://www.explorestemcells.co.uk/TherapeuticCloning.html>.

• "60% PARKINSON’S PATIENTS IMPROVE AFTER REPAIR STEM CELL TREATMENT – PHYSICIANS CONFIRM RESULTS! « The Stem Cell Blog." The Stem Cell Blog. Web. 24 Mar. 2011. <http://repairstemcell.wordpress.com/2010/02/06/60-parkinsons-patients-improve-after-repair-stem-cell-treatment-physicians-confirm-results/>.

• "Pros and Cons of Stem Cell Therapy." Health Guidance - Free Health Articles. Web. 24 Mar. 2011. <http://www.healthguidance.org/entry/12366/1/Pros-and-Cons-of-Stem-Cell-Therapy.html>.

• 24, Posted: March. "Progress Seen in Creating Eye Cells From Stem Cells - US News and World Report." Health News Articles - US News Health. Web. 24 Mar. 2011. <http://health.usnews.com/health-news/managing-your-healthcare/womens-health/articles/2011/03/24/progress-seen-in-creating-eye-cells-from-stem-cells?PageNr=1>.