Universidad Tecnológica de Corregidora

Ingeniería en Biotecnología
BI-802

Inglés VIII

Gene Therapy

Presentan:​ ​ ​ ​ ​ ​ ​ ​ Docente:
Angel Camacho Ricardo​ ​ ​ ​ ​ Ing. Valdez Gómez Andrea
Gene Therapy

Pros of Gene Therapy

Gene therapy is a groundbreaking advancement in medical science that offers hope for individuals
suffering from genetic disorders. One of the most significant advantages is its potential to provide a
permanent cure rather than just treating symptoms. By directly modifying or replacing faulty genes,
patients can achieve long-term relief from diseases such as cystic fibrosis, sickle cell anemia, and certain
types of cancer. Unlike traditional treatments that often require lifelong management, gene therapy aims
to eliminate the disease at its source, improving patient outcomes and reducing suffering. This means
that instead of taking medicine every day, people can receive a single treatment that lasts a lifetime.

Another benefit is its ability to reduce the overall burden on healthcare systems. Since gene therapy
targets the root cause of genetic diseases, it may eliminate the need for lifelong treatments, frequent
hospital visits, and expensive medications. This not only improves the quality of life for patients but also
reduces healthcare costs in the long run. For instance, patients with hemophilia, who require regular
clotting factor infusions, could benefit from a one-time gene therapy treatment that allows their bodies
to produce the necessary proteins naturally. This would free them from the need for constant medical
care and give them more independence.

Furthermore, gene therapy paves the way for medical innovations. It has the potential to treat rare
diseases that previously had no cure, and ongoing research continues to expand its applications. In the
future, gene therapy could be used to treat conditions like Alzheimer's, Parkinson's disease, and even
hereditary blindness, offering a new frontier in personalized medicine. With advancements in genome
sequencing and precision medicine, doctors may soon tailor treatments to individual patients, enhancing
effectiveness and minimizing side effects. This could mean that medicine will be more personalized,
helping each person in a unique way.

Additionally, this technology has ethical implications related to enhancing human life. Gene editing
techniques, such as CRISPR, could prevent genetic disorders before birth, giving future generations a
healthier start in life. By eliminating harmful mutations, society may witness a significant decrease in the
prevalence of hereditary diseases. Preventing conditions such as Huntington’s disease, which is currently
incurable, could drastically improve public health and life expectancy. If parents could choose to remove
harmful genes from their children, it would mean fewer illnesses in the future and a healthier population
overall.

Another key advantage of gene therapy is its potential impact on cancer treatment. Scientists are
exploring gene-editing techniques to reprogram immune cells, enabling them to target and destroy
cancer cells more effectively. This approach, known as CAR-T cell therapy, has already shown promising
results in treating leukemia and lymphoma, paving the way for more effective and less invasive cancer
treatments in the future. Unlike chemotherapy, which can harm healthy cells, gene therapy could attack
only the cancer cells, making treatment more precise and reducing side effects.

Cons of Gene Therapy

Despite its promising benefits, gene therapy is surrounded by ethical, technical, and financial
controversies. One of the primary concerns is the potential for unintended genetic modifications.
Altering the human genome carries risks of unpredictable consequences, including the development of
new genetic disorders or unwanted mutations. Scientists are still working to ensure the safety and
accuracy of gene-editing techniques to minimize these risks. Mistakes in gene therapy could lead to
unforeseen health problems, possibly affecting not only the treated patient but also future generations if
germline editing is involved. If scientists are not careful, they might create new problems instead of
solving old ones.

Another major drawback is the high cost of gene therapy treatments. The development and
implementation of these therapies require significant financial investment, making them inaccessible to
many patients. Some of the currently available gene therapies cost millions of dollars per patient, raising
questions about affordability and equitable access to these life-saving treatments. If only wealthy
individuals can afford gene therapy, it could create a significant healthcare disparity, exacerbating social
inequalities. This means that rich people could have better health while poor people continue to suffer
from diseases, which would not be fair.

Moreover, there are ethical dilemmas regarding the use of gene therapy for non-medical purposes. Some
fear that genetic modifications could lead to the concept of "designer babies," where genetic engineering
is used for aesthetic or performance enhancements rather than medical necessity. This raises concerns
about social inequality and the potential misuse of genetic technology. If gene therapy is used to select
traits such as intelligence, physical appearance, or athletic ability, it could lead to a new form of genetic
discrimination and ethical dilemmas regarding human enhancement. Parents might start choosing how
their children look or how smart they are, which could change society in unexpected ways.

Additionally, the long-term effects of gene therapy remain unknown. Since this field is relatively new,
there is still uncertainty about how genetic modifications might impact future generations. Some
researchers warn that changes made to human DNA could have unforeseen consequences that may not
become apparent for decades. If altered genes are passed down to offspring, it could have unintended
consequences for future populations, potentially leading to new diseases or complications that current
medical knowledge cannot predict. This uncertainty makes many people worry about the risks involved.

Another concern is the regulatory and legal challenges associated with gene therapy. Governments and
medical organizations must establish strict guidelines to ensure that gene-editing technologies are used
responsibly. Without proper oversight, there is a risk that unregulated gene therapies could be
developed and sold without adequate safety testing, putting patients at risk. The lack of standardized
global regulations also raises concerns about "medical tourism," where patients seek unapproved or
experimental treatments in countries with looser restrictions. This could be dangerous because some
people might receive unsafe treatments that could harm them instead of helping them.

Finally, some people believe that gene therapy interferes with nature. They argue that genetic diseases
are part of human life and that trying to change them is unnatural. Others worry that playing with human
DNA could lead to unexpected problems, such as the loss of genetic diversity. If everyone starts removing
"bad" genes, it might lead to new issues that scientists cannot yet predict. Some religious and cultural
groups also oppose gene therapy, believing that altering human DNA is against their beliefs.

In conclusion, while gene therapy holds immense potential for treating genetic diseases and improving
human health, it is accompanied by various challenges. Ethical considerations, financial barriers, safety
concerns, and regulatory issues must be carefully addressed to ensure that this technology is used
responsibly and effectively. Continued research, public discourse, and regulatory oversight will play a
crucial role in shaping the future of gene therapy and its impact on society. If managed correctly, gene
therapy could revolutionize modern medicine, but it must be approached with caution to ensure its
benefits outweigh the risks. This technology has the power to change lives, but it also comes with great
responsibility.