1.

The dilemma that is being faced in the “Bubble Kid Gene Therapy” is that the vector integrated
to the person’s genes are found, but these vectors are inserted in random locations in the
genome and inserts regulatory sequences that might lead to activate the genes near the
genome and might cause cancer that cause a high risk of death.
2. Is there a higher risk of death due to the “Bubble Kid Gene Therapy?”
3. Facts:
a. SCID was the first condition to be treated with gene therapy more than 20 years ago.
b. Severe combined immunodeficiency (SCID) is also known as “bubble boy” disease, since
people affected must live in a sterile environment.
c. Adenosine deaminase is an enzyme that usually dispatches a toxic molecule from white
blood cells. In its absence, the toxin builds up, killing the cells that fight infections.
4. The possible decisions could be:
a. They can risk the life of their loved one even though it is mostly as lose to lose basis.
b. They can trust the process but not expect thoroughly good results afterwards.
c. They can choose whether if they would want their loved ones suffer from its body-based
illness or suffer from a possible scientific based illness.
d. They can use the process but expect, “what is it for my family and me if this wouldn’t be
successful.
5. Additional information are:
a. Earlier this year, three children with a degenerative enzyme disorder were successfully
treated using a modified lentivirus, along with three with an immune disorder called
Wiskott-Aldrich syndrome.
b. An 18-year-old also died following a reaction to a virus used in gene therapy for a liver
condition.
c. But in subsequent trials, four young patients were diagnosed with leukemia two years
after receiving a similar treatment.
d. Promising results have also been seen in degenerative disease adrenoleukodystrophy
and the blood-cell disorder beta-thalassemia. Around 700 gene therapy trials using
lentiviruses are ongoing.
e. Other vectors are showing promise too. For example, adeno-associated virus (AAV)
doesn’t insert its genes into the genome, but places them alongside it, meaning they get
read but are not passed to subsequent generations of cells.
6. Based on weighing out the pros and cons of the said practice, the group chooses to stand
against Bubble kid gene therapy.
7. Ethical Principles:
a. The chances of the success after gene therapy is uncertain.
b. It may create a future where children have their genetic profiles altered in vitro so that a
specific result is created.
c. It may encourage gene doping
 8. Authority that support your claim
Gene therapy has been an issue since 1990, and our group chooses to stand it. There are
many reason why our group stands against it, people tend to play God when it comes to
changing the natural, genetic makeup of a person or a living being and it is ethically wrong,
while curing a disease cause by genetic issues, it can develop a new illness, the results is not that
impressive and only limited number of patient can afford because it is very expensive. According
to the Joint Appeal Against Human and Animal Patenting, a coalition of Protestant, Catholic,
Jewish, Muslim, Buddhist and Hindu Leaders, calling God’s creation as an invention is incorrect,
much of the money that they are using in their research is from the taxpayer and it very
expensive.
9. Formulate a rebuttal
Theoretically, every person has a chance of getting cancer or HIV/AIDS but is it practical
for every person to receive gene therapy? No because Gene therapy is too much expensive.
Gene therapy is yet another aspect of the American lifestyle increasing the gap between the rich
and the poor. These Gene Therapy showed us that limited people can afford this therapy.
People died in experimenting gene therapy because the mortality rate is very high (The test was
for embryotic stem cell for research and within two days of the procedure he suffered from
brain damage and organ failure and went into a coma).

10. Qualifier

Gene therapy insert a working gene into a person with a faulty version that the product should
overcome the defect to integrate the gene into the patient’s DNA for the cell can be persuade. To
overcome this, researchers have turned it into lentiviruses; that still insert gene randomly but can be
modified to disable some regulatory sequence. According to Patrick Aubourg, the new generation of
lentiviral vectors is much safer, although the risk is not zero percent. Around 700 gene therapy trials
using lentiviruses are ongoing, promising a result to have been seen in degenerative disease
adrenoleukodystrophy and the blood cell disorder beta-thalassaemia.

Trials and tribulations of gene therapy

1990 First approved gene therapy trial. Immune cells from 4-year-old Ashanti DeSilva are given working
versions of the ADA gene to treat severe combined immunodeficiency disorder (SCID). It works, but only
temporarily.

1992 ADA-SCID is successfully treated through gene therapy on stem cells harvested from bone marrow.

1999 18-year-old Jesse Gelsinger dies following an immune reaction to the virus vector used to insert
the corrected gene. US Food and Drug Administration suspends several trials pending re-evaluation of
ethical and procedural practices.

2000 Announcement that two boys in France with X-linked SCID or “bubble boy” disease have been
cured using gene therapy.

2002 French SCID trial suspended after four children develop leukaemia as a result of the retrovirus
vector.
2003 Chinese company Shenzhen SiBiono GeneTech gains approval for treating head and neck cancer
with Gendicine, a modified adenovirus carrying a tumour-suppressor gene. Researchers in the US begin
the first human trial using a modified lentivirus (pictured below). It is a disabled HIV virus carrying a gene
to inhibit replication. Trial is a success.

2009 Eight-year-old Corey Haas, who has a rare inherited eye disease and is almost blind, gains normal
vision following gene therapy to replace a retinal pigment protein. Progression of the degenerative
disease adrenoleukodystrophy is halted in two boys using gene therapy.

2010 An adult with blood disorder beta-thalassaemia no longer needs monthly blood transfusions
following gene therapy to insert a corrected beta-globin gene into stem cells that make blood.

2011 Six people with clotting disorder haemophilia B see a reduction in symptoms after gene therapy on
liver cells.

2012 Glybera becomes the first gene therapy drug to be approved in the West, with European approval
to treat lipoprotein lipase deficiency.

2013 Two papers describe the treatment of children with a degenerative disorder called metachromatic
leukodystrophy and immune disorder Wiskott-Aldrich syndrome using gene therapy (Science,
doi.org/pnv; doi.org/ppk).