Assignment on Advantages and Disadvantages of Gene Therapy

1. Assignment on Advantages and Disadvantages of Gene
Therapy.
Course Title: Advanced Pharmaceutical Biotechnology
Course Code: Pharm-6108
Submitted To,
Abdullah Faruque
Professor
Department of Pharmacy
Jahangirnagar University.
Submitted By,
Hridoy Kumer Dey
Roll no: 1808
M. Pharm Examination 2020
Department of Pharmacy
Jahangirnagar University

2. Advantages and Disadvantages of Gene Therapy
Advantages:
• Provides options: Gene therapy can potentially cure someone of a disease, especially
in instances where no other medications have worked.
• Only has to be given one time: Existing gene therapies are given as a one-time dose.
This is appealing compared to some other treatments that require multiple doses.
• Long-lasting effects: Gene therapy has the potential to get rid of a person’s symptoms
for life. Gene therapy can give many people a better quality of life.
• Positive effects passed down through generations: If you remove a faulty gene from
a parent, they won’t transfer this gene to their kids.
• Rapidly-changing technology: Gene therapy is able to exist because of massive
advances in technology over the past few decades. Since technology continues to
advance, we can expect gene therapy to continue to improve. We can also expect more
gene therapies to gain FDA approval.
Disadvantages:
• Expensive: Gene therapy can be extremely pricey, making it inaccessible for some
people. For example, Zolgensma, a gene therapy to treat spinal muscular atrophy, is the
most expensive medication in the U.S. It costs $2.1 million for a course of treatment.
• Experimental: Gene therapy is relatively new and there’s still a lot about it that we
don’t know. Right now, there are no guarantees. Exactly how successful gene therapy
will be is still uncertain.
• Potentially dangerous: There’s some concern that if gene therapy reaches other cells
in the body besides the faulty cell, it could have harmful effects. There’s also concern
that reproductive cells could be changed without detection, and these could be passed
on to people’s children.
• Ethical issues: Some people have ethical issues with gene therapy. For example, some
wonder who will decide what “good” and “bad” uses of gene therapy are? And will
gene therapy make us less accepting of people who are different?
• May cause infection: Some gene therapies use inactivated viruses to carry healthy
genetic material into a faulty cell in the body. It’s possible that these viruses could infect
the person receiving therapy.
• DNA mutations: The new gene might be inserted in the wrong location in the DNA,
which might cause harmful mutations to the DNA or even cancer.
• Viral spread: Because viruses can affect more than one type of cell, it is possible that
the viral vectors may infect cells beyond just those containing mutated or missing
genes. If this happens, healthy cells may be damaged and cause other illness or diseases,
including cancer.