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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.

Adenovirus

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Short answer

Briefly describe the risks associated with somatic cell gene therapy.

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Questions & Answers

what are the two acids the skin produce
Caro Reply
alpha hydroxy and beta hydroxy there are water soluble compounds and often use as exfoliant
Enow
what must a positive strand of an RNA virus do first
Kelsi-Ann Reply
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.
Dejene
 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
Dejene
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.
Dejene
Why strong acid and alkline are not harmful to mycobacterium bacili?
Brian
What are the types of bacteria
John Reply
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.
Melanie
what other characteristics of prokaryotes a bacteria don't have?
Brian
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.
Dejene
gram positive bacteria and gram negative bacteria
mubeen
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.
Dejene
all of you are amazing microbiologists
demisew
thanks demisew....
Dejene
guys what are the two acids the skin produce
Caro
what are the bacteria's involved in the decaying of food
Enow Reply
Some pathogenic bacteria, such as Clostridium perfringens and Bacillus cereus, are capable of causing spoilage.
Dejene
Corona has a gray and black cell structure ....if yes explain..if no explain
Joshua Reply
multiple questions and answers in microbiology and bio chemistry
Lakshmi Reply
is the study of a bacteria and other organisms
isir
yes..this book is about bacteria & others organisms
Hasan
biochemistry is the branch of science that dealing of chemical compounds reactions and other processes
isir
have you any question?
Hasan
yess, Why scientists not search coronavirus vaccines in short time.
demisew
they are on
Monyditchol
what are the symptoms for tuberculosis
Chiamaka
Most people infected with the bacteria that cause tuberculosis don't have symptoms. When symptoms do occur, they usually include a cough (sometimes blood-tinged), weight loss, night sweats and fever.
Dejene
symptoms tuberculosis. Fever Chills Night sweats Cough Loss of appetite Weight loss Blood in the sputum (phlegm) Loss of energy
lourdes
can corona virus transmitted from mother to her child through placenta ?
Abdul
probably
Mad
no
Oke
no,but it can through trait
Falere
mutation occur in the genome of corona virus. thats why the corona vaccines forming just difficult
Muzamil
intersted
Do
no
Esther
No
John
Yes
Suhaib
yes
Benjamin
yes coz it's spread through the soft body parts more so the openings in our bodies
Rebecca
Yes
Ayan
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Abdul
because it damages the immune system by reduction the action of WBC
mike
reducing pls
mike
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Prince Reply
we term it as discharge
Monyditchol
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evans Reply
What is a varuis
evans
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
Ebo
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
Dejene
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.
What are the important of capsules
Marriam Reply
what are the roles of male sex hormones
Marriam
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
Dejene
Capsules in bacteria protect them from phagocytosis of eukaryotic organisms. This is what makes them virulent and harmful without antibodies.
Lewis
any one told me definition of amoebic dysentery & amoebic liver dysentery?
Mira
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....
Dejene
tnx
Mira
u welcome mira
Dejene
people explain for me this words in public health.tb prevention 1:promotive 2:preventive 3:curative 4:rehabilitative
Obrian
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.
Melanie
...As some antibotics focus on breaking down the cell wall and is not able to.
Melanie
what are the clinical classification of amoxicillin?
Rebecca
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Oliver Reply
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
Lewis
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Mohamed Reply
Anatomy is the study of parts of the human body
Matilda
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.
Dejene
Describe the halden effect
Suleiman
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.
Dejene
Difference between chief cells and parietal cells in the stomach
Suleiman
 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.
Dejene
is the study of structure and organs located in human life
isir
listen to Matilda
Lewis
essay on microbiology and how it contribute to the pharmacy assistant programme
Tagedevi Reply
I want to know how it contribute to the pharmacy assistant programme
chidiebube
Contribute how? If you want to contribute to pharmaceutical stuff you should look for websites with blogs that relate to your interests.
Lewis
hello i want to know how it contribute to microbiology programs
Dejene
Microbiology is the study of bacteria and and organisms such as viruses, fungi, and mold. How does this apply to medicine? It applies to medicine or pharmacology because when you get sick you are infected by a pathogen and understanding how these organisms interact with each other helps you to....
Melanie
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.
Melanie
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.
Melanie
what is a bacterial
Eric Reply
Bacteria is a microscopic organism belonging to the kingdom prokaryotic
John
what is prokaryotic
Oliver
if you are here, read this free book, it is mostly correct, there are a few pictures that should be corrected
Lewis
thanks
Eric
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.
Eric
HIV And Coronavírus, what is the difference ? Both are Retrovirus, please , I want to know. Thank you.
Cl Reply
does it mean Corona is incurable just like the HIV for the it to be retroviral too?
Joy
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
Dejene
***niaid.nih.gov/diseases-conditions/coronaviruses start here, since this is what is new, then recap yourself on autoimmune diseases that are not recoverable
Lewis
what happen when virus escape the host
Hailemichael
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.
Dejene
what is the difference between capsule and glycocylx in relation to prokaryotes
Felix Reply
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.
Dejene
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
assarra
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
assarra
Meu I watch at microscopic HIV
Cl
assarra is paying attention. look into taking a course is you want to truly understand what is known now
Lewis

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Source:  OpenStax, Microbiology. OpenStax CNX. Nov 01, 2016 Download for free at http://cnx.org/content/col12087/1.4
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