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Ethical concerns

Beyond the health risks of gene therapy, the ability to genetically modify humans poses a number of ethical issues related to the limits of such “therapy.” While current research is focused on gene therapy for genetic diseases, scientists might one day apply these methods to manipulate other genetic traits not perceived as desirable. This raises questions such as:

  • Which genetic traits are worthy of being “corrected”?
  • Should gene therapy be used for cosmetic reasons or to enhance human abilities?
  • Should genetic manipulation be used to impart desirable traits to the unborn?
  • Is everyone entitled to gene therapy, or could the cost of gene therapy create new forms of social inequality?
  • Who should be responsible for regulating and policing inappropriate use of gene therapies?

The ability to alter reproductive cells using gene therapy could also generate new ethical dilemmas. To date, the various types of gene therapies have been targeted to somatic cells, the non-reproductive cells within the body. Because somatic cell traits are not inherited, any genetic changes accomplished by somatic-cell gene therapy would not be passed on to offspring. However, should scientists successfully introduce new genes to germ cells (eggs or sperm), the resulting traits could be passed on to offspring. This approach, called germ-line gene therapy , could potentially be used to combat heritable diseases, but it could also lead to unintended consequences for future generations. Moreover, there is the question of informed consent, because those impacted by germ-line gene therapy are unborn and therefore unable to choose whether they receive the therapy. For these reasons, the U.S. government does not currently fund research projects investigating germ-line gene therapies in humans.

Risky gene therapies

While there are currently no gene therapies on the market in the United States, many are in the pipeline and it is likely that some will eventually be approved. With recent advances in gene therapies targeting p53, a gene whose somatic cell mutations have been implicated in over 50% of human cancers, Zhen Wang and Yi Sun. “Targeting p53 for Novel Anticancer Therapy.” Translational Oncology 3 , no. 1 (2010): 1–12. cancer treatments through gene therapies could become much more widespread once they reach the commercial market.

Bringing any new therapy to market poses ethical questions that pit the expected benefits against the risks. How quickly should new therapies be brought to the market? How can we ensure that new therapies have been sufficiently tested for safety and effectiveness before they are marketed to the public? The process by which new therapies are developed and approved complicates such questions, as those involved in the approval process are often under significant pressure to get a new therapy approved even in the face of significant risks.

To receive FDA approval for a new therapy, researchers must collect significant laboratory data from animal trials and submit an Investigational New Drug (IND) application to the FDA’s Center for Drug Evaluation and Research (CDER) . Following a 30-day waiting period during which the FDA reviews the IND, clinical trials involving human subjects may begin. If the FDA perceives a problem prior to or during the clinical trial, the FDA can order a “clinical hold” until any problems are addressed. During clinical trials, researchers collect and analyze data on the therapy’s effectiveness and safety, including any side effects observed. Once the therapy meets FDA standards for effectiveness and safety, the developers can submit a New Drug Application (NDA) that details how the therapy will be manufactured, packaged, monitored, and administered.

Because new gene therapies are frequently the result of many years (even decades) of laboratory and clinical research, they require a significant financial investment. By the time a therapy has reached the clinical trials stage, the financial stakes are high for pharmaceutical companies and their shareholders. This creates potential conflicts of interest that can sometimes affect the objective judgment of researchers, their funders, and even trial participants. The Jesse Gelsinger case (see Case in Point: Gene Therapy Gone Wrong ) is a classic example. Faced with a life-threatening disease and no reasonable treatments available, it is easy to see why a patient might be eager to participate in a clinical trial no matter the risks. It is also easy to see how a researcher might view the short-term risks for a small group of study participants as a small price to pay for the potential benefits of a game-changing new treatment.

Gelsinger’s death led to increased scrutiny of gene therapy, and subsequent negative outcomes of gene therapy have resulted in the temporary halting of clinical trials pending further investigation. For example, when children in France treated with gene therapy for SCID began to develop leukemia several years after treatment, the FDA temporarily stopped clinical trials of similar types of gene therapy occurring in the United States. Erika Check. “Gene Therapy: A Tragic Setback.” Nature 420 no. 6912 (2002): 116–118. Cases like these highlight the need for researchers and health professionals not only to value human well-being and patients’ rights over profitability, but also to maintain scientific objectivity when evaluating the risks and benefits of new therapies.

  • Why is gene therapy research so tightly regulated?
  • What is the main ethical concern associated with germ-line gene therapy?

Key concepts and summary

  • While gene therapy shows great promise for the treatment of genetic diseases, there are also significant risks involved.
  • There is considerable federal and local regulation of the development of gene therapies by pharmaceutical companies for use in humans.
  • Before gene therapy use can increase dramatically, there are many ethical issues that need to be addressed by the medical and research communities, politicians, and society at large.

Fill in the blank

_____________ is a common viral vector used in gene therapy for introducing a new gene into a specifically targeted cell type.


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Short answer

Briefly describe the risks associated with somatic cell gene therapy.

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Questions & Answers

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A positive-sense single-stranded RNA virus (or (+)ssRNA virus) is a virus that uses positive sense single stranded RNA as its genetic material. Single stranded RNA viruses are classified as positive or negative depending on the sense or polarity of the RNA.
 The positive-sense viral RNA genome can serve as messenger RNA and can be translated into protein in the host cell. Positive-sense ssRNA viruses belong to Group IV in the Baltimore classification. Positive-sense RNA viruses account for a large fraction of known viruses, including many pathogens
such as the hepaci virus C, West nail virus, dengue virus, SARS and MERS coronaviruses, and SARS-CoV-2 as well as less clinically serious pathogens such as the rhinoviruses that cause the common cold.
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Do you mean the shapes or the the two different types of bacteria? Bacteria are often described in terms of their general shape. Common shapes include spherical (coccus), rod-shaped (bacillus), or curved (spirillum, spirochete, or vibrio) The two different types are gram negative or gram positive.
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Prokaryotes are single-celled organisms belonging to the domains Bacteria and Archaea. Prokaryotic cells are much smaller than eukaryotic cells, have no nucelus, and lack organelles. All prokaryotic cells are encased by a cell wall. Many also have a capsule or slime layer made of polysaccharide.
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Most bacteria can be broadly classified as Gram positive or Gram negative. Gram positive bacteria have cell walls composed of thick layers of peptidoglycan.cells stain purple when subjected to a Gram stain procedure. Gram negative bacteria have cell walls with a thin layer of peptidoglycan.
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Some pathogenic bacteria, such as Clostridium perfringens and Bacillus cereus, are capable of causing spoilage.
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A submicroscopic infectious organism, now understood to be a non-cellular structure consisting of a core of DNA or RNA surrounded by a protein coat. It requires a living cell to replicate, and often causes disease
A virus is a biological agent that reproduces inside the cells of living hosts. When infected by a virus, a host cell is forced to produce thousands of identical copies of the original virus at an extraordinary rate
A virus is a microorganism which invade our bodies causing diseases due to eliciting immune responses by the body against it, can replicate using our genome inducing production of proteins helping them to establish new life inside our bodies.
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Testosterone is the principal sex hormone inmales and is produced in the testes (testicles). Dihydrotestosterone is a hormonein which the double bond of testosterone has been reduced by enzyrne reactions in the body. ... The testes perform two functions: They produce sperm, and they producetestoster
Capsules in bacteria protect them from phagocytosis of eukaryotic organisms. This is what makes them virulent and harmful without antibodies.
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Amoebiasis, also known amoebic dysentery, is an infection caused by any of the amobae of the Entamoeba group. Symptoms are most common during infection by Entamoeba histolytica. Amoebiasis can be present with no, mild, or severe symptoms. Symptoms may include abdominal pain, diarrhea....
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Capsules function similarly to endospores they provide an extra layer of protection especially in acidic or basic environments. It is also a thicker membrane which can change the osmosis process and can provides resistance to antibotics depending if it is gram negative or positive.
...As some antibotics focus on breaking down the cell wall and is not able to.
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simply autoimmune disease is not completely understood. There are many variations from genetically inherited to acquired by viruses like HIV. Genetically they may not be prominent until an unknown point in one's life. I am far from an expert, I am just reciting what I have learned. Take rheumatoid
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Anatomy is the study of parts of the human body
the branch of science concerned with the bodily structure of humans, animals, and other living organisms, especially as revealed by dissection and the separation of parts.
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The Haldane effect is a property of haemoglobin first described by John ScottHaldane. Oxygenation of blood in the lungs displaces carbon dioxide from hemoglobin which increases the removal of carbon dioxide. This property is the Haldane effect.
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 Parietal cells are the epithelialcells that secrete HCl and intrinsic factor. They are located in the gastric glands found in lining of fundus and stomach. The gastric chief cells , are cells in the stomach that release pepsinogen and chymosin.
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develop medicine. A lot of bacteria infections can be cured with various medicines but not all medicines work equally. It depends if your sickness is based on gram positve or negative bacteria, if its s mold or fungus or a virius. Each medicine targets a certain one.
If you need any ideas I recommend looking up Louis Pastar who used microbiology to invent a lot of medicines and contributed greatly to microbiology and pharmaceutical.
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Bacteria is a microscopic organism belonging to the kingdom prokaryotic
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A prokaryotes does not have lipid- bilayer bound organelles, they can reproduce by binary fission, they have a DNA region, most have a cell well, contains a plasmid, 70s ribosomes, high mutation rate due lack of certain DNA replication enzymes.
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The reason researchers are looking to these medicines is that the new coronavirus—like HIV, Ebola, and hepatitis C—are all RNA viruses.but covid-19 is to detect human and animal virus. hiv is only human virus do not detecte animals
***niaid.nih.gov/diseases-conditions/coronaviruses start here, since this is what is new, then recap yourself on autoimmune diseases that are not recoverable
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A virus must attach to a living cell, be taken inside, manufacture its proteins and copy its genome, and find a way to escape the cell so that the virus can infect other cells. Viruses can infect only certain species of hosts and only certain cells within that host.
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However glycocalyx exists in bacteria as either a capsule or a slime layer. Thedifference between a capsule and a slime layer is that in a capsule polysaccharides are firmly attached to the cell wall, while in aslime layer, the glycoproteins are loosely attached to the cell wall.
Glycocalyx is composed of glycosaminoglycans, proteoglycans and other glycoproteins bearing acidic oligosaccharides and terminal sialic acids. Capsule is Composed of polysaccharide (i.e. poly: many, saccharide: sugar). Exception: The capsule of Bacillus anthracis is composed of polymerized D-glutami
Capsule is located immediately exterior to the murein (peptidoglycan) layer  of gram-positive bacteria and the outer membrane (Lipopolysaccharide layer) of gram-negative bacteria The glycocalyx, which is located on the apical surface of endothelial cells, is composed of a negatively charged network
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