SCIENCE, TECHNOLOGY, AND SOCIETY:

Gene Therapy for Severe Combined Immune Deficiency

Presented to Science, Technology, and Society Teacher

Mrs. Genalyn Cuence - Ocay

In partial fulfillment of the requirements in

SCIENCE, TECHNOLOGY, AND SOCIETY

BY DOCTOR OF DENTAL MEDICINE 1

SECTION MESIODENS GROUP 8

ROSCA, EGIE BOY

SABIDO, MARK KARYLLE
SANTIAGO, CLINT DAVID
SICAT, GIAN FALCO
SURIBA, MARTINA REGINE

November 25, 2022

Gene Therapy for Severe Combined Immune Deficiency

Gene Therapy is a medical technique that has created huge advancements in

the medical field by treating diseases which are caused by a change in a person’s

genes. Through use of genetic material in treating and preventing diseases, gene

therapy has a potential to give hope for those with fatal diseases, stop any damage from

occurring within an individual, and can design treatments that targets any genetic

material that causes the disease. Although it has its challenges and risks such as a

significantly large amount of time that is needed in order to find the right diagnosis or

potential treatments, no actual guarantee of it working, and the uncertainties that go

along with it, many of those who have undergone gene therapy have had successful

cases.

One of these successful cases was the Severe Combined Immune Deficiency

also known as SCID. It was the first ever gene therapy trial in which the first pioneer

was Dr. William French Anderson in the year 1990. Although its first trial was

successful, it was short-lived. After further investigation and research, in 2002 they were

able to create a successful breakthrough and achieve full recovery in this genetic

deficiency.

Severe Combined Immune Deficiency is an inherited immune system disease

known as "primary immunodeficiency." It is a collection of uncommon illnesses brought

on by mutations in several genes involved in the growth and operation of immune cells

that fight infections. It is caused by gene defects that affect the function of T cells and B

cells, and NK cells can also be affected depending on the type of SCID. These cells
play a critical role in helping the immune system battle bacteria, viruses, and fungi that

cause infections. Severe Combined Immune Deficiency is extremely rare, occurring in

about 1 in 100,000 births. Symptoms of SCID are similar to those of other childhood

illnesses, such as sinus, respiratory infections that keep coming back, ear infections,

overgrowth of yeast in the mouth, severe diaper rash, skin infections that don't respond

well to treatment, diarrhea, and slow growth or weight gain. Children with SCID are

more prone to infections than others and they may need to be physically isolated to

protect them from disease. There's no guarantee that the body will clear these infections

when treated, which can be life-threatening.

Newborn screening are tests that frequently look for disorders that might impede

newborns growth, including hearing issues, particular heart difficulties, genetic and

metabolic abnormalities, and other conditions. Development of these tests has made it

possible to detect SCID before symptoms appear, ensuring that affected infants receive

life-saving treatments. Because infants with SCID have few or no T cells, the absences

of TRECs may indicate SCID. To confirm a SCID diagnosis, doctors will evaluate the

numbers and types of T and B cells present and their ability to function. Research

supported by “National Institute of Allergy and Infectious Diseases” (NIAID) and other

organizations, newborn screening for SCID can result in an early diagnosis, rapid

treatment, and high survival rates.

Hematopoietic stem cell (HSC) gene therapy is the standard treatment for infants

with SCID. Ideally, infants with SCID receive stem cells from a sibling who is a close
tissue match, because transplants from matched siblings result in the best recovery of

immunological function. Infants may also get stem cells from a parent or an unrelated

donor if a matched sibling is not available. Although these transplants can save lives,

they frequently only partially restore immunity.

Overall, gene therapy is one of the treatments that made considerable

advancements in the medical field. It alters the genetic code to treat diseases to correct

any underlying genetic problem such as the Severe Combined Immune Disease (SCID).

Gene therapy can be dangerous however this treatment gives other people a chance to

live their lives normally.

What are the pros and cons of genetically modifying crops?

By significantly altering patterns of herbicide use toward glyphosate, which is

thought to be a relatively benign herbicide in this regard, GM crops may have a positive

impact on human health by reducing exposure to insecticides. Be that as it may, these

cases are for the most part founded on suspicion as opposed to genuinely exploratory

information. Any public work that has been done to date has been met with skepticism

and criticism, including Seralini's work. There are generally no public studies on the

potential health effects of eating food or feed derived from GM. The Food and Drug

Administration in the United States and the European Food Safety Authority in Europe,

however, have granted regulatory approval to the commercialized GM crops as safe for

human consumption. Multivitamin corn and maximum zeaxanthin corn, both of which

hold great promise but have not yet been commercialized, are examples of the
improvement of GM crops that will have a direct impact on health. These crops will have

lower levels of allergens, higher levels of protein and carbohydrates, and higher levels

of essential amino acids, essential fatty acids, vitamins, and minerals.

GM crops have also been criticized for encouraging the growth of pests that are

resistant to pesticides. The overreliance on a single pesticide and the overuse of a

limited number of pesticides are the primary causes of resistant pests' development.

This would be especially true for glyphosate because glyphosate use was very limited

prior to the development of Roundup Ready crops, and there has been an explosion of

glyphosate-resistant weed species since Roundup Ready crops were introduced.

Cross-pollination with wild pests (weeds) results in the development of resistant pests,

which is frequently cited as a major problem, but this is much less of a concern because

it is extremely unlikely. However, there are issues when genes transfer from GM crops

to non-GM crops, resulting in unexpected volunteer crops that are resistant to

herbicides, which can present challenges for farmers and cost them money. In

conclusion, GM crops are both advantageous and disadvantageous in the world of

agriculture.

What are the pros and cons of gene therapy?

The advantage of the gene therapy technique is giving someone born with a

genetic disease or developing cancer a chance to live an everyday life. This technique

can cure many diseases that have affected society for years. It is an advantage that far

outweighs any disadvantages presented by gene therapy. People opposed to gene

therapy need to understand the technique. In the future, gene therapy will play an
essential part in many people's lives. Gene therapy will revolutionize the practice of

medicine. The role of doctors and patients will change in the coming years, and it will all

be due to gene therapy. In addition, gene therapy is going to be a significant

advancement for science. However, gene therapy also has its disadvantages, the

body’s immune system might see the various viruses that we use to replace unwanted

genes as invaders that must be extinguished before they cause harm. When the white

blood cells attack the newly introduced genetic material, it is not unusual for a patient to

experience health issues like inflammation, dizziness, and headaches. In severe

reactions, it is even possible for the immune response to target the body’s organs and

cause them to fail.

References:

20 Advantages and Disadvantages of Gene Therapy. (2019, August 18).

FutureofWorking.com. https://futureofworking.com/6-advantages-and-

disadvantages-of-gene-therapy/

‌Acker, R. van. (2017). Pros and Cons of GMO Crop Farming. Oxford Research

Encyclopedia of Environmental Science.

https://oxfordre.com/environmentalscience/view/10.1093/acrefore/978019938941

4.001.0001/acrefore-9780199389414-e-217

‌Gene Therapy Basics | ASGCT - American Society of Gene & Cell Therapy |. (n.d.).
Retrieved November 23, 2022, from https://patienteducation.asgct.org/gene-
therapy-101/gene-therapy-basics
Gene Therapy Successes. (2012). Utah.edu.
https://learn.genetics.utah.edu/content/genetherapy/success/
Human Gene Therapy. (2022). Ndsu.edu.

https://www.ndsu.edu/pubweb/~mcclean/plsc431/students/zhou.html

Patel, H. M. (2019). Gene Therapy for Severe Combined Immunodeficiency

(SCID). News-Medical.net. https://www.news-medical.net/health/Gene-Therapy-

for-Severe-Combined-Immunodeficiency-(SCID).aspx

Severe Combined Immunodeficiency Symptoms, Diagnosis & Treatment. (2022).

Aaaai.org. https://www.aaaai.org/Conditions-Treatments/Primary-

Immunodeficiency-Disease/severe-combined-immunodeficiency

Severe Combined Immunodeficiency (SCID). (2019). NIH: National Institute of

Allergy and Infectious Diseases.

https://www.niaid.nih.gov/diseases-conditions/severe-combined-

immunodeficiency-scid

Treating the bubble babies: gene therapy in use. (2021). Yourgenome · Science

Website. Retrieved November 23, 2022, from

https://www.yourgenome.org/stories/treating-the-bubble-babies-gene-therapy-in-

use/