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Introduction to Cloning
Cloning is a biotechnology tool that uses the genes of a biological species to create an identical copy. In the past scientists have cloned basic cells and genes, and even complex organisms such as sheep. A complex organism is a multicellular organism with many different types of cells such as skin, blood, or liver cells. The copy that is created from the original is called a clone. Cloning takes away a part of the cultural inheritance that humans achieve through the transmission of experiences and history (Ayala) but it allows for revolutionary forms of medicine to be invested in and studied. Even if cloning an entire organism is controversial, cloning specific cells allows for gene therapies to be created that will allow for the standard of life for millions of people to be increased. Some theories even believe that genetic engineering would allow humans to cure hereditary disease (Singh).
The concept of cloning began in 1938 with Hans Spemann but failed to make strong strides until the 1980s. Soon after the discovery of DNA, in 1978, David Rorvik released a book titled In His Image: The Cloning of a Man that began a controversial debate on the ethics of cloning. Revolutionizing the concept of cloning genetic data and healthy cells was life-saving because looking for perfect match bone marrow donors was difficult. In the early 1970s, the concepts of gene therapy were raised (NIH). It advanced to researchers manipulating viruses in order to deposit the DNA for the desired gene to insert genetic material into the virus (NIH). The first human trial was in 1990 for a four-year old girl who needed help with treatment against her genetic disease. It was successful and the cells were placed to provide longer-lasting benefits for her.
This research paper will further expand on the process of somatic cell gene therapy and how exactly it works technically and ethically. Somatic cell gene therapy places genes in the somatic cells and aims to cure diseases in that person, not their descendants. I will also focus on the problems or dangers of gene therapy and any side effects or negative facets it might have. Understanding specific genetic disorders and gene therapy methods that are being studied, this paper will also focus on cystic fibrosis specifically and how gene therapy has helped create treatments that could lead to a long term treatment that solves the genetic disease.
Goals of Cloning
One of the main purposes of cloning is to create healthy and functioning stem cells, because they repair, build, maintain our body and therefore can be manipulated to help repair tissues and organs in the body. In the past, stem cells have been transferred through bone marrow transplants but those result in lots of failed results where the body’s immune system does not accept the transplant. Cloning would allow for genetically identical stem cells for each individual. In fact, In 2013, scientists at Oregon Health and Science University were the first to use cloning techniques to successfully create human embryonic stem cells (Learn Genetics Utah).
In order to create identical genetic material, scientists have focused on gene cloning. Gene cloning specifically focuses on producing copies of genes or segments of DNA (Cloning Fact Sheet). It has a completely different process than reproductive or therapeutic cloning. With gene cloning, my research will specifically focus on its benefits with gene therapy, including using specific genes to create biopharmaceuticals targeted to help people. For centuries humans have had genetic diseases that only recently were identified but were still unable to be cured. Gene therapy is an unprecedented science because it is a form of cloning that is revolutionary in its goals and methods.
Specifically, gene therapy involves altering the genes inside your bodys cells in an effort to treat or stop disease (Mayo Clinic Staff). Mutated genes can cause disease so research and advancements in gene therapy allows doctors and scientists to try to create a normal copy of a gene that is mutated. It can assist people with disabilities by trying to fix nonfunctional genes that do not perform the tasks the healthy human body is supposed to. If conducted properly, gene therapy is defined as the introduction of genetic material via techniques of molecular biology into somatic cells [in contrast to germ cells] to treat or prevent disease. (McCain).
Replacing a mutated gene allows for cells that have become diseased due to genes that do not work correctly to then be able to start helping to treat certain diseases they could not fight before. For example, A gene called p53 normally prevents tumor growth and if it is replaced where it is missing then it can cause cancer cells to be eliminated (Mayo Clinic Staff). Fixing mutated genes inhibits diseases because the mutation is, in a sense, turned off . Doctors and scientists would also be able to use gene therapy in order to condition a persons immune system to recognize and attack diseased cells.
The specific procedure of gene therapy is done through clinical trials that are conducted in different ways. Clinical trials help scientists test whether certain medicines or practices are safe to use on people. To complete a specific type of gene therapy includes blood or bone marrow being drawn from a large needle, exposing that sample to a virus that contains the desired genetic material, and that virus being injected back into a persons body with the altered genes (Mayo Clinic Staff). A gene is inserted into a cell that does not function through a vaccine or IV by using a virus as a vector in order to deliver the gene. (NIH). Once inserted, the virus disassembles and delivers the genetic data into the nucleus and they are modified not to create diseases but rather to restore the function of the protein that is malfunctioning. (NIH).
By adding a new gene if one is missing or replacing a mutated one, people born with defects such as being unable to fight disease or having a disability will be helped. Gene therapy is relatively new and since 2000 has already proven helpful in helping create long and short term solutions for cystic fibrosis, X-SCID disease, and more. The development of gene therapy will allow for replacement of defective genes, alteration of an aberrant gene, or introducing pharmaceuticals in genes that can assist with the eradication or treatment of a disease. So far, gene therapy has shown to be successful in treating diseases such as leukemia, blindness, hemophilia, and immune deficiency (Mayo Clinic Staff).
Cloning Applied to Cystic Fibrosis
Cystic Fibrosis is an autosomal-recessive genetic disease that causes lung infections and limits the ability for a person to breathe (CFF). Mutations in the Cystic Fibrosis Transmembrane Conductance (CFTR) gene causes the protein to not be able to move chloride to the cell surface to attract water and therefore the mucus in the lungs clogs the airways and traps germs leading to complications (CFF). Gene therapy is revolutionary with cystic fibrosis because it takes correct copies of the CFTR DNA to the epithelial cells in the airways (Burney and Davies). Without It is still a difficult process because Cystic Fibrosis is a life-long disease and therefore the procedure has to be repeated multiple times. Currently, scientists are working on the clinical efficacy of Cystic Fibrosis gene therapy treatment so that the mutation can be solved long term for human beings.
Cloning Risks & Regulations
Risks included with gene therapy circle around the fact that the gene usually has to be manipulated by inserting it into a virus which has risks. The original diseases-causing genes are removed but your bodys immune system may see the newly introduced viruses as intruders and attack them (Mayo Clinic Staff). Other risks also include viruses targeting the wrong cells, such as healthy cells which would damage them. The virus can also cause infection due to unpredictability of exactly how it will react, sometimes even leading as far as causing a tumor if it is inserted incorrectly into the wrong spot in a persons DNA. The risks are due to the modernity of the science behind gene therapy, the techniques are fairly new so institutions such as the U.S. Food and Drug Administration continue to regulate research. Although there has been almost miraculous work performed with gene therapy, there are still many developments that need to be made particularly in the areas of gene delivery and cell transplantation in order for it to be replicated and performed on a larger scale (Mulligan).
The ethical issues that revolve around gene therapy continue to place the safety of the research in the highest regard, but that results in a slower movement of the innovations with gene cloning. The main ethical problems regarding gene cloning include distinguishing the moral line between good and bad, drawing a line between the disabilities and normal traits that gene therapy hopes to fix, and the costs and access associated with gene therapy. It also jumps into a cosmetic arena when discussing which traits should be enhanced or fixed with gene therapy such as height, intelligence, or athletic ability (NIH). Long term side effects of gene therapy have not been studied in depth due to how new it is so there are unexpected risks associated with targeting embryonic cells. Therefore, in the United States, the government does not allow federal funds to be used for research on germline gene therapy (NIH). Organizations such as the NIH and FDA continue to limit and monitor the frontlines of research with cloning and gene therapy.
Strictly discussing full body human cloning has many ethical implications. Homosapiens have been naturally created since the beginning of their existence, and to conjure a fully formed one with cloned embryonic cells would result in consequences that can not be predicted. First off, humans are raised with cultural influences that give them their background, morals, virtues, dreams, passions, memories, and experiences. Creating a human that is already an adult would take those abstract experiences away and that human being would not be able to understand the societal aspects of life. Other ethical issues are that humans would not be able to know if the clone feels pain, can be destroyed, their life spans, and how they are mentally, emotionally, and physically defected by their created. Regulations that limit the research of human cloning are important because they also require scientists at the forefront of cloning research to be ethical in their actions and not create beings for immoral reasons.
In the United States, there are currently no federal laws outlawing cloning. Rather, the laws address the ethical rules regarding the funding towards cloning research. Different states do directly outlaw certain forms of cloning. These issues rose after the famous cloning of Dolly the Sheep and Congress created bills that outlawed human cloning to somatic cells, pursuing research that would allow a complete human to be cloned, and placing a cloned fetus into a womans uterus (The New Atlantis). Yet, due to the first amendment, there is no law that directly outlaws cloning. The German, Canadian, and Italian governments have directly outlawed cloning and have strict laws in place. The United Nations has been a strong voice in coercing member nations to create strong restrictions that limit the research of human cloning due to the ethical codes it would violate.
The Future of Cloning
In addition to expanding the knowledge base in cellular, developmental, and molecular biology, as well as in cancer and aging, cloning has now been applied to enhance medicine and agriculture (Berardino). Scientists are continuing to study the development of cells during meiosis and how somatic cells are differentiated. This research leads to the hope that mice, livestock, and endangered species will be able to be produced through cloning (Trounson). Creating patient-specific embryonic stem cells would continue to revolutionize the face of medicine because it would allow for unpredictable genetic diseases to be cured and the standard of life for human beings that would have otherwise had short life expectancies or had to live in pain to be increased.
This alone makes the research of cloning worth being continued because already it is helping the lives of so many people be bettered and creating ways to solve problems with animal endangerment that otherwise was not possible. In the future, ethical concerns remain regarding the reproduction of humans but the laws in place will limit unethical creations. Cloning will allow for skin cells that are damaged on a human being through accidents or illness can be replaced. If it is studied it will continue to create unique, genetically programmed cells that can cure diseases such as Parkinsons or Cystic Fibrosis.
Bibliography
- Ayala, F. (2015). Cloning Humans? Biological, ethical, and social considerations. Proceedings of the National Academy of Sciences of the United States of America. 112(29) doi: 10.1073/pnas.1501798112
- About Cystic Fibrosis. (n.d.). Retrieved from https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/
- Cloning Fact Sheet. (2017, March 21). National Human Genome Institute. Retrieved from https://www.genome.gov/about-genomics/fact-sheets/Cloning-Fact-Sheet
- Berardino, M. A. (n.d.). Cloning: Past, Present, and the Exciting Future. Retrieved from https://www.faseb.org/Portals/2/PDFs/opa/cloning.pdf
- Burney TJ, Davies JC. Gene therapy for the treatment of cystic fibrosis. Appl Clin Genet. 2012;5:2936. Published 2012 May 29. doi:10.2147/TACG.S8873
- Friedmann, T. (1989). Progress toward human gene therapy. Science, 244(4910), 12751281. doi: 10.1126/science.2660259
- How does gene therapy work? – Genetics Home Reference – NIH. U.S. National Library of Medicine (n.d.). Retrieved from https://ghr.nlm.nih.gov/primer/therapy/procedures
- Lee, T. W. R., Matthews, D. A., & Blair, G. E. (2005). Novel molecular approaches to cystic fibrosis gene therapy. Biochemical Journal, 387(1), 115. doi: 10.1042/bj20041923
- McCain J. (2005). The future of gene therapy. Biotechnology Healthcare, 2(3), 5260.
- McKinnell, G., R., Berardino, D., & A., M. (1999). Biology of Cloning: History and Rationale. BioScience 49(11) Retrieved from https://academic.oup.com/bioscience/article/49/11/875/220023
- Mulligan, R. C. (1993, May 14). The basic science of gene therapy. 260(5110) Retrieved from https://science.sciencemag.org/content/260/5110/926
- Part Four: Cloning Policy in the United States. (n.d.). Retrieved from https://www.thenewatlantis.com/publications/part-four-cloning-policy-in-the-united-states
- Singh, D. (2004, February 28). Human cloning is justified in preventing genetic disease. 328(7438) Retrieved from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1125280/
- Trounson A.O. (2006) Future and Applications of Cloning. In: Verma P.J., Trounson A.O. (eds) Nuclear Transfer Protocols. Methods in Molecular Biology
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