Gene Therapy

(STEM CELL THERAPY

XENOTRANSPLANTATION )

Reporters:
Delloro, Khiel Ross
Ejercito, Josanovhem Q.
Luage, Kem
Sumaylo, Jhapoy
Sambaan, Meljane
Gene Therapy

- Gene therapy is the next generation of medicine targeting the underlying cause of a
genetic disease. It has the potential to offer patients a transformational clinical benefit
and dramatically improve quality of life.

What are genes?

- A gene is the basic physical and functional unit of heredity, and is made up of
DNA.They are passed from parent to child.

- Genes are the blueprints providing instructions on how to make proteins for the body.
Proteins play an important role in how our body functions.

- These genes control everything from hair color, to height, to many other traits. As far
as we know, humans have between 20,000 and 25,000 genes.

Genetic Disorder

- Sometimes there is a mutation, a change in a gene or genes.

- The mutation changes the gene's instructions for making a protein, so the protein does
not work properly or is missing entirely.

Three types of genetic disorders:

• Single-gene disorders, where a mutation affects one gene. Sickle cell anemia is an
example.

• Chromosomal disorders, where chromosomes (or parts of chromosomes) are missing

or changed. Chromosomes are the structures that hold our genes. Down syndrome is a
chromosomal disorder.

• Complex disorders, reflects a disorder that results from the contributions of multiple
genomic variants and genes in conjunction with significant influences of the physical
and social environment.

How Gene Therapy Work?

- This new genetic material, such as a working gene, is delivered into the cell using a
vector. A vector is like a package used to deliver a specific message.
The vector can be delivered in one of two ways:

1. EX-VIVO treatment- Removes the person’s own cells and delivers the genetic
material to these cells outside the body. The modified cells are then returned to
the body.
2. IN-VIVO treatment- Genetic material is delivered directly into the person, such as
through an injection.

History of Gene Therapy

Gene therapy 1.0: First introduction of corrected genes

- As early as the 1960s, scientists speculated that DNA sequences could be introduced
into patients’ cells to cure genetic disorders.

Gene therapy 2.0: Improved viral vectors

- Scientists developed better viral vectors to deliver genetic therapies. In the early
1980s, David Williams, MD, and David Nathan, MD, at Boston Children’s Hospital
published the first paper showing one could use a virus to insert genes into
blood-forming stem cells.

Gene therapy 3.0: Gene editing and Base Editing

- Gene editing can knock out a faulty gene, insert a new DNA sequence, or both in a
“cut and paste” operation. Base editing is even more fine-tuned. It leverages the
targeting ability of CRISPR, but relies on enzymes to chemically change one “letter” of a
gene’s code at a time — changing, say, C to T or A to G.

FDA-Approved Gene Therapies

Abecma (idecabtagene vicleucel)

- treat multiple myeloma, a type of cancer related to your bone marrow.

Imlygic (talimogene laherparepvec)

- treat a type of skin cancer called melanoma.

Zynteglo (betibeglogene autotemcel)

- treats severe beta-thalassemia, a genetic blood disorder.

Luxturna (voretigene neparvovec-rzyl)

- treats retinal dystrophy, an inherited condition that affects your retinas.

POTENTIALS

1. Hope for fatal disease.

2. Earlier can be better.

3. Target the cause

DISADVANTAGES

1. Long-term effects unknown

2. Repeat Administration

3.No guarantees

STEM CELL THERAPY

Stem cells - are special to human cells that are able to develop into many different cell
types. This can range from muscle cells to brain cells.

Two Types of Stem Cells:

1. Adult Stem Cell - are undifferentiated cells found throughout the body after
development. These cells are essential in the daily healing, growth, and
replacement of cells lost.
2. Embryonic Stem Cell - are derived from an early stage of human development,
typically from the blastocyst stage of an embryo. They are pluripotent, meaning
they have the potential to differentiate into any cell type of the body.

Stem cell therapy

- also known as regenerative medicine, promotes the repair response of diseased,

dysfunctional or injured tissue using stem cells or their derivatives. It is the next chapter
in organ transplantation and uses cells instead of donor organs, which are limited in
supply.

- Stem cells can be administered in a variety of fashions; IV Stem Cell Therapy

(intravenous administration), Intrathecal (directly into the spinal canal), stem cell
injections into problem areas (knee, hips, hands, etc.).
FDA-Approved (STEM CELL Therapies)

Laviv (azficel-T)

- help treat moderate to severe cosmetic wrinkles near your mouth and nose.

Provenge (sipuleucel-T)
- treat advanced prostate cancer.

Omisirge (omidubicel-onlv)
- helps adolescents and adults with blood cancer regain white blood cells during the
stem cell transplant process.

MACI (autologous cultured chondrocytes)

- placed inside your knee to help with cartilage growth.

POTENTIAL
1. Minimum post-procedural recovert
2. Less to no risk rejection
3. Helps avoid surgery and its risks of complications
4. A potential way to reverse an injury

DISADVANTAGES
1. Misleading Marketing
2. Potential for adverse reaction
3. Ethical Considerations
4.Lack of Regulation
 GENE THERAPY VS STEM CELL THERAPY

GENE THERAPY
- Introduction or modification of genes to treat or prevent disease.
- This usually involves a virus vector to deliver the new gene.
- Gene therapy involves the transfer of genetic material in a carrier or vector and the
uptake of the gene into targeted cells.
- Introduction of cells to repair or replace the damaged cells.
- Some stem cells (mesenchymal stem cells) can act as vectors.
- Cell therapy involves the transfer of cells with relevant/desired function into the patient.

STEM CELL THERAPY

- Introduction of cells to repair or replace the damaged cells.
- Some stem cells (mesenchymal stem cells) can act as vectors.
- Cell therapy involves the transfer of cells with relevant/desired function into the patient.

XENOTRANSPLANTATION

INTRODUCTION
Human organ transplantation is a relatively new field of medicine that is now facing a
significant challenge. Because of its clinical success, the need for this procedure far
exceeds the availability of donor organs. One solution doctors along with
pharmaceutical and biotechnology companies are investigating to end this acute
shortage is "Xenotransplantation."

XENOTRANSPLANTATION

- is when non-human cells, tissues, or organs are used to treat medical conditions in
humans.

- xenotransplantation is an experimental treatment. It is only allowed in extremely rare

and serious cases. The FDA has not yet allowed clinical trials to begin. These trials will
be important to show if this procedure is truly safe and effective.
HISTORY OF XENOTRANSPLANTATION

1. The first experiments in transplanting chimpanzee kidneys into humans were

conducted in 1963 and 1964. One of the patients who received chimpanzee kidneys
lived for nine months.

2. In the last two decades involved Baby Fae, the infant who received a baboon heart in
1984, and Jeff Getty, an AIDS patient who received a bone marrow transplant from a
baboon in 1995.

3. Researchers are currently experimenting with pigs as sources of organs and tissues
for xenotransplantation. Studies include the use of pancreatic islet cells and neural cells
from pigs for insulin-dependent diabetes and refractory parkinsonism.

WHAT ANIMAL IS COMMONLY SUBJECT FOR DONOR?

Pigs have become the most commonly used animal for xenotransplantation of organs
into humans. Their early sexual maturity (5 months), short gestation periods (3.5
months), large litters (5-10 piglets) and suitable organ size, offer advantages over other
animals. Pig tissues (such as routinely used heart valves) have been successfully
transplanted into people.

THE DONOR

The pigs used for this procedure are raised in a laboratory for this purpose. Their genes
are edited to better match human genes. This helps lower the risk of rejection in
humans. Rejection is when the body attacks the new organ.

POTENTIAL

1. Provide an unlimited supply of cells, tissues, and organs for humans

2. Prompt surgery

3. Liberal age limits

DISADVANTAGES

1. Organ rejection

2. Risk of infection

3. Ethical issues

CONCLUSION
Genetic therapies, stem cell therapies and xenotransplantation hold promise to treat
many diseases. All approaches are offering great and valuable treatment options for
devastating and life-threatening diseases and recent advances have made genetic
therapies much safer and accessible.

The said therapies has the potential to be to uplift the power and capability of
technologies in helping reach the society a quality and longer lives we seek for.
REFERENCES

1.MedlinePlus. (n.d.). Genetic Disorders. Retrieved from

https://medlineplus.gov/geneticdisorders.html

2.Advanced Sports and Spine. (n.d.). 10 Benefits of a Stem Cell Treatment. Retrieved
from https://www.advancedsportsandspine.com/10-benefits-of-a-stem-cell-treatment/

3.Mayo Clinic. (n.d.). Bone Marrow Transplant: Stem Cells. Retrieved from
https://www.mayoclinic.org/tests-procedures

4.Children's Hospital Answers. (n.d.). Gene Therapy History. Retrieved from

https://answers.childrenshospital.org/gene-therapy-history/

5.Pfizer. (n.d.). Genes as Medicines. Retrieved from

https://www.pfizer.com/science/innovation/gene-therapy/genes-as-medicines#:~:text=G
ene%20therapy%20is%20a%20technology,a%20defect%20in%20one%20gene%E2%8
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