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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

introduction to microbiology
Shweta Reply
Study the life of microorganisms present in the environment their survivals etc..
ayesha
Microscopic organisms, commonly known as microorganisms or microbes, are found all around us and even inside our bodies. The category 'Microbes' includes a massive range of organisms including bacteria, fungi, viruses, algae, archaea and protozoa.
Amre
fermented foods likely benefited ancestors to preserve foods and make other foods like milk, cheese, and bread using microbes.
Zinnia Reply
the evidence that I would support the statement regarding ancient people ideas that disease was transmitted by things they could not see is by the discoveries under a microscope. Many microbiologists have discovered certain diseases caused by microbes.
Zinnia
plz give the information about glyoxylate cycle
kamini Reply
what type of information?
Sapiens
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Kisota
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Sapiens
what is microbial soup?
Osborn Reply
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ayesha
most bacterial pathogens that cause disease in humans are classified in terms if their preferable ph as
Beesan Reply
what is micro
Kabul Reply
extremely small in size
Swetha
almost invisible to the naked eye
Peter
thanks
Sadiq
uwc
Peter
invisible sized objects
Sivasri
invisible sized object or specimens.which is used to see in only the microscope that specimens or objective called the micro
Sivasri
small organism that can be seen by use of Microbes
Manyang
why do you need to study microorganisms
Sala
what is microbiology?
Green Reply
microbiology is a branch of biology .it deals with the study of microorganisms life cycle , uses, disadvantages,and it impact in any other fields .which is used to view in microscope .
Sivasri
Microbiology is the study of microorganisms, those being unicellular, multicellular, or acellular. Microbiology encompasses numerous sub-disciplines including virology, bacteriology, protistology, mycology, immunology and parasitology. 
Raj
In short , it is the study of micro organisms and types , characteristics.
Kaviya
can you explain the structure of rna in detail
Dhanalakshmi Reply
RNA is of many types....like mRNA,tRNA,rRNA,snRNA,guideRNA..etc .
Swetha
but generally RNA is single stranded
Swetha
It contains adenine guanine cytosine , instead of thymine it contains uracil.
Swetha
ribonucleic acid present in RNA.. where as in DNA it is deoxyribonucleic acid...and ribonucleotide is present in RNA
Swetha
RNA is a blue print of DNA. it has the information from DNA....and we can predict the base panirs in DNA if we have the RNA....copy of that DNA
Swetha
can either have positive or negative polarity
Ernestine
Diatoms need..... With the help of which they can construct their beautiful cell wall
Prathmesh Reply
Microbial growth curve shows a.... Curve.
Shambhuraj Reply
diminishing curve
Oluwapamilerin
It represents the decreasing growth of an organism.
Swetha
what is meant by reverse isolation in tuberculosis treatment?
Swetha Reply
why do we study microbiology
HABIBA Reply
Microbiology is the way of gaining knowledge about those organisms which are surround us but we can't see them with our naked eye,such organisms called microorganisms..We can see them only under microscope.
ayesha
from our birth to death microorganisms are with us in the environment as well as on our body which are called Microflora.
ayesha
Microfloras are non pathogenic
ayesha
what is microbiology
Anji Reply
It is the study of microorganisms and their structures functions and their interactions with environment ...
Swetha
it is the study of microorganisms which are invisible to backed eye but we can see them under microscope then microbiology is the study of microorganisms including their structures , functions,and their interactions with environment
Swetha
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Anji
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Swetha
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Anji
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Anji
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Swetha
from India
Swetha
which state
Anji
Hyderabad .
Swetha
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Anji
chelusu nakku kochum
Swetha
thank you
Swetha
what is reverse isolation in tuberculosis treatment?
Swetha
Reverse isolation is used to protect you from germs when your immune system is not working properly. Germs can be carried on droplets in the air, medical equipment, or another person's body or clothing.
Kaviya
will you repeat this again?
Swetha
Reverse isolationis used to protect you from germs when your immune system is not working properly. Germs can be carried on droplets in the air, medical equipment, or another person's body or clothing.
Kaviya
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Swetha
Y
Kaviya
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Swetha
What is allosteric site?
Hetshree Reply
I don't know how to explain but it's a deep concave site of an enzyme it's slightly or more slightly similar to the active site on an enzyme
ayesha
a site other than enzyme active site
Muhammad
describe the gram staining procedure as used in identification and classification of bacteria and it's interpretation
tabitha Reply
Gram staining method found by Hans Christian Gram. Classified into Gram Positive and Gram Negative Positive would be purple or blue stained bacteria Negative would be Pink or Red stained bacteria Both are observed under microscope This procedure performed on glass slides by making a smear of sample.
ayesha
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shamim
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ayesha
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shamim
Masha allah
Khamis
jazakallah Khair
ayesha
hi
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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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