Semi- Finals

Science Technology and Society

Name: Neil Jeff C. Panis Code: GE 411

1. Discuss how Gene therapy works.

How Gene Therapy Works

1. Gene therapy aims to address the underlying cause of disease,

such as changes in our genes. If genes are like the blueprint to our
body, gene therapy can fill in missing parts or correct errors in the
drawings.

Gene therapy is the use of genetic material to treat or prevent

disease. The genetic material that is delivered, DNA or RNA, has
instructions to change how a protein—or group of proteins—is
produced by the cell. For some diseases, this means making
changes to account for too much, not enough, or incorrect essential
proteins being produced within cells.

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. Viruses can be used as vectors because they have
evolved to be very good at getting into cells. Scientists have learned
how to remove the viral genes and use this same ability to treat or
prevent disease. In this case, their goal is to insert the new genetic
material into the cell. All viral vectors are tested many times for safety
prior to being used in humans.

The vector can be delivered in one of two ways:

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.

in-vivo treatment means the genetic material is delivered

directly into the person, such as through an injection.

https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-ba
sics

2. Gene therapy works by altering the genetic code to recover the

functions of critical proteins. Proteins are the workhorses of the cell
and the structural basis of the body’s tissues. The instructions for
making proteins are carried in a person’s genetic code, and variants
(or mutations) in this code can impact the production or function of
proteins that may be critical to how the body works. Fixing or
compensating for disease-causing genetic changes may recover the
role of these important proteins and allow the body to function as
expected.

Gene therapy can compensate for genetic alterations in a couple

different ways.

Gene transfer therapy introduces new genetic material into cells. If an

altered gene causes a necessary protein to be faulty or missing, gene
transfer therapy can introduce a normal copy of the gene to recover
the function of the protein. Alternatively, the therapy can introduce a
different gene that provides instructions for a protein that helps the
cell function normally, despite the genetic alteration.

Genome editing is a newer technique that may potentially be used for

gene therapy. Instead of adding new genetic material, genome editing
introduces gene-editing tools that can change the existing DNA in the
cell. Genome editing technologies allow genetic material to be added,
 removed, or altered at precise locations in the genome.
CRISPR-Cas9 is a well-known type of genome editing.

https://medlineplus.gov/genetics/understanding/therapy/procedures/

2. Discuss the Potentials, Challenges and Risks of Gene Theraphy.

Potential, Challenges, & Risks of Gene Therapy

1. Potential
Hope for fatal disease. Gene and cell therapy can help treat diseases
that have limited treatment options. Without treatment many of these
inherited disorders would end in severe disability or premature death.
But with gene and cell therapy, early studies show that these
disorders have been slowed or completely stopped.

Earlier can be better. If gene therapy is received earlier in the course

of disease, it has the potential to stop any damage before it occurs. It
is still being researched to what extent a gene therapy might reverse
any damage.

Targets the cause. Gene and cell therapies make it possible to design
treatments that can target any of the thousands of genes in the body.

2. Challenges
Time. It is a lengthy process, often many years, to test potential
treatments in clinical trials and undergo the FDA approval process. It
may also take time to find a diagnosis for a particular gene variation
causing disease symptoms.

Not one-size-fits all. Each disease with a certain gene variation

requires a specific gene therapy approach. Currently, most gene and
cell therapy options are limited to treating only the disorders that are
caused by variations in a single gene. People may have changes in
different genes but still have the same disease. In this situation, they
would likely need different gene therapy approaches each targeted to
their gene variation. Work is being done to develop platforms and
standards that will speed the development and delivery of customized
gene therapies. One example is the Bespoke Gene Therapy
Consortium, among many others.

Small number of participants. Gene therapy often targets rare

diseases, meaning there is a small number of people with the
disease. This means it can take longer or be more difficult to gather
enough research data on potential treatments or to find enough
people that are eligible to participate in a clinical trial. Although each
disease may be rare, rare diseases combined affect an estimated
400 million people. There are numerous hard-working patient
advocacy groups and foundations working to fund and move research
along in this space.

Funding. There are high costs that go into the work it takes to prepare
a clinical trial, make a gene therapy, and ensure it is carried out at the
highest standard and ensuring safety for participants.

Accuracy required. Gene and cell therapies need to express the gene
in the right tissue, at the right level, for the right amount of time. This
means that a lot of research goes into the best way to deliver the
genetic material. The response of the person’s immune system also
needs to be considered based on the therapy.

3.Risks
Informed consent. Before participating in a clinical trial, a member of
the research team should review any potential risks and benefits with
the participant and/or caregiver. It is important that participants
understand their rights during the research process and know what to
expect.

No guarantees. Therapies being studied in clinical trials are not a

guaranteed cure and cannot guarantee beneficial results. There is
always a chance that the investigational treatment may not work or
may have unexpected side effects.

Repeat administration.The delivery of foreign material to the body can

cause an immune response and may exclude patients from receiving
later treatments. Participating in a clinical trial may also prevent future
participation in other trials or from additional types of treatments. This
includes if a better therapy is developed later on.

Long-term effects unknown. Gene therapy can be an alteration for a

lifetime, so people should be aware that there could be long term
effects (both good or bad) that are unknown at this time. That is why
gene therapies are monitored in a person for years following
treatment.

https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-ba
sics