The Nano World and Gene Therapy

At present, advances in science and technology have become more apparent. Inventions and
new technologies transformed the world and the everyday lives of every one of us. Many things
that were mere visions of the future yesterday are now a reality. But of course, there are still a
lot of tech innovations that remain as a goal.

Overview

Over the past few decades, research has been moving towards the infinitely small- the
nanoworld. The production of nanotechnologies and nanodevices is now occurring at a rapid
pace. Its applications are widely diverse and have affected a variety of fields of science and
industry including energy, environmental science, food security, electronics, and medicine.

In medicine, nanotechnology promises to revolutionize drug delivery, diagnostic techniques,

and gene therapy. Scientists develop new gene therapy approaches that offer a promise for one
day treating different inherited and acquired diseases.

We have seen the huge potentials of new technologies or fields of study, but how about the
risks? It is important for us to examine the potential for unintended consequences, especially
those related to human and environmental health.

https://education.nationalgeographic.org/resource/nano -world

The Nano World

Just as human understanding of the natural world was revolutionized by the discovery of light
microscopes, modern microscopes that can expose and change individual atoms are once again
exposing a whole new world-the nano world. Scientific researchers have developed new
technological tools that greatly improved different aspects of our lives through the use of
nanoscale.

Nanotechnology refers to the science, engineering, and technology conducted at the nanoscale.
It is concerned with building 'things' - generally, materials and devices on the scale of atoms
and molecules. It encompasses science and technology that manufactures materials of great
help to the improvement of various areas of society especially health, environment, energy,
electronics, food, water, and agriculture.

Nanomanufacturing

It refers to scaled-up, reliable, and cost-effective manufacturing of nanoscale materials,

structures, devices, and systems. It leads to the development of new products and improved
materials. Below are the two fundamental approaches to nanomanufacturing.
1. Bottom-up fabrication- It manufactures products by building them up from atomic-
and molecular-scale components. However, this process can be time-consuming. Scientists and
engineers are still in search for effective ways of putting up together molecular
components that self-assemble.

2. Top-down fabrication- It trims down large pieces of materials into nanoscale. This
process needs larger amounts of materials and discards excess raw materials.

What is the difference between bottom-up fabrication and top-down fabrication?

Top-down approaches are good for producing structures with long-range order and for making
macroscopic connections, while bottom-up approaches are best suited for assembly and
establishing short-range order at nanoscale dimensions.

Bottom-up fabrication uses chemical and physical forces at a nanoscale level to assemble
simple units into larger structures. The bottom-up approach mimics biological processes, where
individual atoms pile up one after the other on a substrate to form molecules.

Top down fabrication can be likened to sculpting from a block of stone. A piece of the base
material is gradually eroded until the desired shape is achieved. That is, you start at the top of
the blank piece and work your way down removing material from where it is not required.
Benefits and Concerns of Using Nanotechnology

Nanotechnology has various applications in different sectors of the society and environment.
However, there are concerns that need to be addressed before using and promoting materials
derived from nanotechnology.

1. Nanotechnology is not a single technology; it may become prevalent.

2. It seeks to develop new materials with specific properties.

3. New efficiencies and paradigms may be introduced that may render certain natural
resources and current practices uncompetitive or obsolete.

4. It may be complicated to detect its presence unless one has the specialist tools of
nanotechnology.

Benefits and Concerns of the Application of Nanotechnology in Different Areas

Example of Areas Affected by Possible Benefits Concerns
Nanotechnology Possible
Benefits Concerns
Environment • Improved detection and • • High reactivity and toxicity
removal of contaminants Pervasive distribution in
• Development of benign • the environment
industrial processes and No nano-specific EPA
materials regulation
Health • Improved medicine • Ability to cross cell
membranes and
translocate in the body
• No FDA approval needed
for cosmetics or
supplements
Economy • • Better products • • Redistribution of wealth
New jobs Potential cost of cleanups
and healthcare
• Accessibility to all income
levels.
Gene Therapy

Medical Science has detected many human diseases related to defective genes. These types of
diseases are not curable by traditional methods like taking readily available medicines.

Gene therapy is an experimental technique for the treatment or prevention of disease using
genes. In the future, instead of using drugs or surgery, this technique could allow doctors to
treat a disease by inserting a gene into the cells of a patient.

The Basic Process

There are several approaches to gene therapy. These are the following:

• Replacement of mutated gene that causes disease with a healthy copy of the gene.
• Inactivation of a mutated gene that is functioning improperly.
• Introducing a new gene into the body to help fight a disease.

In general, a gene cannot be directly inserted into a human gene or cell. A gene is inserted into
another gene using a carrier or vector. At present, the most common type of vectors are viruses
that have been genetically changed to carry normal human DNA.

Two Types of Gene Therapy https://www.yourgenome.org/facts/what-is-gene-

therapy

1. Somatic gene therapy- involves the manipulation of genes in any cells of the body
except sperm and egg cells that will be helpful to the patient but not inherited to the patient's
descendants.

2. Germ-line gene therapy- involves the genetic modification of reproductive cells such
as sperm and egg cells that will pass the change on to the next generation.

Stem Cell Gene Therapy

Have you heard about stem cells in the news? If yes, perhaps you've wondered if they might
help you or your loved one with a serious disease. You may wonder how they're being used to
treat diseases, and why they are being discussed so vigorously.

Stem cells are mother cells that have the potential to become any type of cell in the body.
Under the right conditions in the body or a laboratory, stem cells divide to self-renew or
multiply. They can become cells of the blood, heart, bones, skin, muscles, brain, among others.

Why is there such an interest in stem cells? Researchers and doctors hope stem cell studies can
help to:
1. Increase understanding of how diseases occur.

2. Generate healthy cells to replace diseased cells (regenerative medicine).

3. Test new drugs for safety and effectiveness.

Sources of stem cells

1. Embryonic stem cells- are derived from a three to five-day-old human embryo that
is in blastocyst phase of development and has about 150 cells. The embryos are usually extras
that have been created in IVF (in vitro fertilization) clinics where several eggs are fertilized in a
test tube then implanted into a woman.

2. Somatic stem cells- are cells that exist throughout the body after embryonic
development. They are also capable of self-renewal and, with appropriate signals,
differentiate into various cell types from the organ from which they are derived. The
extent to which they are capable of creating various types of cells is controversial.

The Bioethics of Gene Therapy

There are ethical issues involved in gene therapy. Some of the inquiry cited are the
following:

1. How can “good” and “bad” uses of gene therapy be distinguished?

The same as good vs bad therapy of any kind. Good therapy benefits the patient, bad
therapy harms the patient. This is an outcome based definition. It may be difficult to know
ahead of time what the outcome may be. I know of at least one notorious instance where a
patient with a non-life-threatening condition was killed by attempted gene therapy.

There are gray areas. Successful therapy usually corrects a defect, but the defectiveness of
some differently abled conditions is a matter of controversy. For example, there is a school
of thought that does not recognize deafness as a disability. Different varieties of cognitive or
emotive ability would be another area where irreversible therapies should be approached
with caution — although I hasten to add that in general the genetic contribution to such
conditions is not well enough understood at present to even contemplate genetic
intervention.

2. Who decides which traits are normal and which constitute a disability or
disorder?

“Normal” is a statement of how common something is. It’s not actually related to whether
something was a disorder.
 The determiner of whether something is a disability/disorder is down to whether the medical
or psychological community believes it is a defect - if it is a net lowering of a capacity,
rather than an increase (Genius isn’t normal, but it’s not a disability) or a different approach.

The problem comes because support only really exists for disability/disorder, rather than
difference, which incentivizes a disability focused approach, where people look for deficits to
make something disabling so the difference can be supported.

It’s why there’s a lot of controversy over autism, for example. It’s not a disability - it’s a
fundamentally different way of approaching the world, which is not inherently deficit. (You
wouldn’t say non-autism was a disability because some non-autists have learning
difficulties!). But if that becomes the model, all the support, all the funding, will dry up.

3. Will the high costs of gene therapy make it available only to the wealthy?

For now, yes indeed. Like any new technology it’s going to be expensive, part of it is
because the treatment itself is expensive but most of the cost is because the companies
that patented these treatments are trying to return their investment and of course, profit,
while they can. Are they justified prices? Probably not.

In this kind of industry patents expire after about a decade, which is when prices go down
to more affordable values, so companies try to profit from their discoveries as much as
possible in that time-frame. It’s likely they’re still going to have high price tags, unless
technology changes or improves in the meantime (which it will).

4. Should people be allowed to use gene therapy to enhance human traits such
as height, intelligence, or athletic ability?

Yes, though only once we actually understand how all the genes interact with each
other. At the moment we only have a very basic understanding what a few genes do. Until
we know enough to build the full genetic sequence from scratch, we've no business playing
around with them outside of experimentation.

References Textbooks:
1. Serafica, et.al. 2018. Science, Technology and Society. Rex Book Store, Inc. 856 Nicanor
Reyes Sr. St., Manila.

2. Doria, et.al. 2018. Science, Technology and Society. Jimczyville Publications. #16
Concha St., Bgry. Tinajeros, Malabon City.

Online References: 1. https://www.nationalgeographic.org/media/nano-world/

2. https://www.medicalnewstoday.com/articles/244972

3. https://www.azonano.com/article/

4. https://www.nano.gov/nanotech-101/what/nano-

5. https://medlineplus.gov/genetics/understanding/therapy/genetherapy/

6.https://www.mayoclinic.org/tests-procedures/bone-marrow-transplant/in-depth/stemcells/art-
20048117

7. https://www.nature.com/scitable/content/embryonic-and-somatic-stem-cells-as-a-60673/

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