Question
Gene Therapy
Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains of fetuses in the womb.
The treatment, which has never been attempted before, would involve doctors injecting the fetus’s brain with a harmless virus that delivers molecules that correct the genetic faults. Tests suggest that the therapy will be most effective around the second trimester, when their brains are in the early stages of development.
The therapy is aimed at a rare brain disorder known as Angelman syndrome, which affects one in 15,000 births. Children with the condition have small brains and often experience seizures and problems with walking and sleeping. They can live their whole lives without speaking a word. Recently, a similar gene therapy treatment was used to stop severe seizures in a newborn, before the child left the hospital.
Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease. Researchers are investigating several ways to do this, including:
1. Replacing mutated genes. Some cells become diseased because certain genes work incorrectly or no longer work at all. Replacing the defective genes may help treat certain diseases. The gene called p53 prevents tumor growth and several types of cancer have been linked to problems with the p53 gene. If doctors could replace the defective p53 gene, that might trigger the cancer cells to die.
2. Fixing mutated genes. Mutated genes that cause disease could be turned off so that they no longer promote disease, or healthy genes that help prevent disease could be turned on so that they could inhibit the disease.
3. Making diseased cells more evident to the immune system. In some cases, your immune system does not attack diseased cells because it does not recognize them as intruders. Doctors could use gene therapy to train your immune system to recognize the cells that are a threat.
A recent survey conducted in England revealed the approval rates for gene therapy applications:
Somatic indicates modification of the genes in body cells, germ-line means that either egg or sperm cells would be genetically modified, and in utero indicates that genetic modifications would take place before birth, while the developing child is in the womb.
What conclusion is supported by the data?
Responses
A Overall, respondents were in favor of genetic modification to treat serious health conditions.Overall, respondents were in favor of genetic modification to treat serious health conditions.
B Respondents were least in favor of genetic modification in utero for either of the three conditions.Respondents were least in favor of genetic modification in utero for either of the three conditions.
C Respondents were no in favor of gene therapy to treat any conditions other than life threatening conditions.Respondents were no in favor of gene therapy to treat any conditions other than life threatening conditions.
D Respondents had no objections to altering somatic cells, but did not approve of altering either sex cells or fetuses in utero.Respondents had no objections to altering somatic cells, but did not approve of altering either sex cells or fetuses in utero.
Question 2
The British survey went on to discovered the hesitation of the survey participants in viewing gene therapy as a viable alternative to traditional methods of treating disease or inherited disorders. Select ALL that could be of concern.
A. The side effects and/or risks of gene therapy are still relatively unknown.
B. Altering the genetic make-up of an unborn child would be in effect, playing God.
C. Who would regulate the practice; government, doctors, big pharma, religious groups?
D. When alternative treatments are viewed as effective and relatively free of side-effects, should gene therapy be considered?
Responses
A BB
B A and BA and B
C B and CB and C
D All could be possible concerns.
Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains of fetuses in the womb.
The treatment, which has never been attempted before, would involve doctors injecting the fetus’s brain with a harmless virus that delivers molecules that correct the genetic faults. Tests suggest that the therapy will be most effective around the second trimester, when their brains are in the early stages of development.
The therapy is aimed at a rare brain disorder known as Angelman syndrome, which affects one in 15,000 births. Children with the condition have small brains and often experience seizures and problems with walking and sleeping. They can live their whole lives without speaking a word. Recently, a similar gene therapy treatment was used to stop severe seizures in a newborn, before the child left the hospital.
Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease. Researchers are investigating several ways to do this, including:
1. Replacing mutated genes. Some cells become diseased because certain genes work incorrectly or no longer work at all. Replacing the defective genes may help treat certain diseases. The gene called p53 prevents tumor growth and several types of cancer have been linked to problems with the p53 gene. If doctors could replace the defective p53 gene, that might trigger the cancer cells to die.
2. Fixing mutated genes. Mutated genes that cause disease could be turned off so that they no longer promote disease, or healthy genes that help prevent disease could be turned on so that they could inhibit the disease.
3. Making diseased cells more evident to the immune system. In some cases, your immune system does not attack diseased cells because it does not recognize them as intruders. Doctors could use gene therapy to train your immune system to recognize the cells that are a threat.
A recent survey conducted in England revealed the approval rates for gene therapy applications:
Somatic indicates modification of the genes in body cells, germ-line means that either egg or sperm cells would be genetically modified, and in utero indicates that genetic modifications would take place before birth, while the developing child is in the womb.
What conclusion is supported by the data?
Responses
A Overall, respondents were in favor of genetic modification to treat serious health conditions.Overall, respondents were in favor of genetic modification to treat serious health conditions.
B Respondents were least in favor of genetic modification in utero for either of the three conditions.Respondents were least in favor of genetic modification in utero for either of the three conditions.
C Respondents were no in favor of gene therapy to treat any conditions other than life threatening conditions.Respondents were no in favor of gene therapy to treat any conditions other than life threatening conditions.
D Respondents had no objections to altering somatic cells, but did not approve of altering either sex cells or fetuses in utero.Respondents had no objections to altering somatic cells, but did not approve of altering either sex cells or fetuses in utero.
Question 2
The British survey went on to discovered the hesitation of the survey participants in viewing gene therapy as a viable alternative to traditional methods of treating disease or inherited disorders. Select ALL that could be of concern.
A. The side effects and/or risks of gene therapy are still relatively unknown.
B. Altering the genetic make-up of an unborn child would be in effect, playing God.
C. Who would regulate the practice; government, doctors, big pharma, religious groups?
D. When alternative treatments are viewed as effective and relatively free of side-effects, should gene therapy be considered?
Responses
A BB
B A and BA and B
C B and CB and C
D All could be possible concerns.
Answers
GPT-4o mini
### Question 1
**What conclusion is supported by the data?**
The correct response is:
**A. Overall, respondents were in favor of genetic modification to treat serious health conditions.**
This option suggests a general approval for serious conditions, which aligns with the context given about gene therapy applications. The other options either imply disapproval or specific concerns that are not clearly supported by the data as stated.
### Question 2
**Select ALL that could be of concern.**
The correct responses are:
**D. All could be possible concerns.**
Each of the options (A, B, C, and D) presents valid points that could contribute to the hesitation regarding gene therapy as a viable alternative to traditional methods.
- **A.** Side effects and/or risks being unknown is a common concern in new medical treatments.
- **B.** The ethical consideration of "playing God" is a frequently discussed ethical dilemma in genetic modifications.
- **C.** Regulation effectiveness and who oversees these practices is a crucial logistical concern.
- **D.** The premise of comparing effectiveness and side effects with alternative treatments is a valid point for consideration.
Therefore, all provided options represent credible concerns regarding gene therapy.
**What conclusion is supported by the data?**
The correct response is:
**A. Overall, respondents were in favor of genetic modification to treat serious health conditions.**
This option suggests a general approval for serious conditions, which aligns with the context given about gene therapy applications. The other options either imply disapproval or specific concerns that are not clearly supported by the data as stated.
### Question 2
**Select ALL that could be of concern.**
The correct responses are:
**D. All could be possible concerns.**
Each of the options (A, B, C, and D) presents valid points that could contribute to the hesitation regarding gene therapy as a viable alternative to traditional methods.
- **A.** Side effects and/or risks being unknown is a common concern in new medical treatments.
- **B.** The ethical consideration of "playing God" is a frequently discussed ethical dilemma in genetic modifications.
- **C.** Regulation effectiveness and who oversees these practices is a crucial logistical concern.
- **D.** The premise of comparing effectiveness and side effects with alternative treatments is a valid point for consideration.
Therefore, all provided options represent credible concerns regarding gene therapy.