Pros and Cons of Gene Therapy

Gene therapy is a medical approach for treating or preventing diseases by correcting any underlying genetic issue. It enables physicians to treat disorders by altering a patient’s genetic makeup rather than relying on surgery or drugs. Early gene therapy methods, gene transfer or addition, introduced new genes into cells to fight diseases, or added a gene’s non-faulty copy to substitute the altered copy bringing the disease.

Advanced studies have led to the discovery of genome editing, a gene therapy technique that initiates molecular tools to alter a cell’s existing DNA. Gene therapy aims at returning an abnormal DNA to a normal condition. This could imply making faulty genes inactive, replacing them with healthy copies, or introducing completely new genes to treat a medical issue. Here are the pros and cons of gene therapy.

Pros of gene therapy

1. It brings hope

Birth defects, including brain or spine, eye, heart, mouth or face, stomach or intestine, muscle or bone, and chromosome defects, affect one in every 33 born in the United States each year, which is about 3% of all babies. Gene defects like trisomy 13 (Patau syndrome) affect 513 babies, trisomy 18 (Edwards syndrome) affects 1,187 babies, and trisomy 21 (Down syndrome) affects 5,568 babies.

These birth defects are a leading cause of infant mortality rates, with 4,000 infants dying yearly. While families struggle to alter these conditions by moving across different healthcare levels, their efforts and hopes are crushed because there’s no cure for such situations. However, gene therapy promises to correct some of these birth defects to ensure children safely grow into their prime.

2. It’s an effective treatment method

Gene therapy successfully treats diseases like neuromuscular disorders, cancer, and blindness. While the success is most qualified, some treatments have effectively alleviated the condition. Some infants born with serious vision loss resulting from retinal diseases which once caused inevitably complete blindness can now benefit from gene therapy by having their vision restored. Through the chimeric antigen receptor (CAR) T cell therapy approach, gene therapy can help program patients’ immune cells to identify and target cancerous mutation cells.

Gene editing is a promising gene therapy for treating blood disorders like sickle cell disease and beta-thalassemia resulting from heritable, single-gene mutations. Gene therapy can also derail potentially lethal illnesses like spinal muscular atrophy (SMA), a neurodegenerative disease where the muscles controlling and those connecting the spinal cord to organs and muscles deteriorate, malfunction, and eventually die.

3. The therapy effects are timeless and long-lasting

Remission chances are limited when defective genes are replaced with functional ones in diseases such as cystic fibrosis. Usually, this treatment is one-off, which sees the patient symptom-free for a lifetime. Additionally, gene therapy isn’t just a solution for the person suffering from a specific condition. It also covers the whole generation. When a gene predisposing someone to breast cancer is removed, they’ll transfer the new functional genes to their children, not the defective ones.

4. Gene therapy is technology-based

Gene therapies exist due to significant technological advancements, and we can expect more therapies to gain FDA approval as technology continues to grow. While gene therapies may be costly at the moment, technological advancements will bring more treatment options, meaning prices will most likely decrease.

5. Gene therapy isn’t limited to humans

IL-12-based gene therapy is a new and effective cancer therapy approach in veterinary medicine. Studies on big animals demonstrate that this therapy approach is effective as systemic treatment via intramuscular therapeutic gene delivery or local intratumoral gene transfer, targeting the disseminated disease or accessible tumor nodules. With several non-viral and viral gene delivery techniques, an excellent local anti-tumor effect has been attained in multiple dogs, horses, and cats tumors, upgrading and supporting several preclinical study results featuring the IL-12-based gene therapy.

IL-12-based gene therapy is an effective and safe therapeutic process that exerts an IFN-y ‘s encoded induction and protein’s systematic release and local transgene response. This makes it a reassuring treatment for big animals whose tumors occur spontaneously.

Cons of gene therapy

While gene therapy can potentially treat several diseases and improve the quality of life, it’s a relatively new approach, and several issues may arise. Here are the cons of gene therapy.

1. It’s costly

While gene therapies are an effective treatment, they’re usually unaffordable to those needing them the most. Drug development and manufacturing costs significantly impact gene therapy’s price tags. The raw materials utilized in gene therapy can be costly based on the illness. In addition, gene therapy technologies aren’t cost-effective. 

Gene therapy prices range from $373,000 for a single dose of CAR-T therapy Yescarta to $2.1 million for Zolgensma. Additionally, these costs cater for the therapies alone- complications, hospital stays, and other medications can potentially increase the treatment costs.

2. The results aren’t guaranteed

The earliest gene therapy studies indicated that it could have severe health risks, including inflammation, cancer, and toxicity. While researchers have continuously improved the treatment techniques, gene therapy is still new and in the experimental stages, meaning risks could be unpredictable.

Since gene therapy is still at a developmental level, anything can go wrong, including death, limiting, or worsening your condition. Immune response and incompatibility issues may result in the procedure’s failure. However, research is still ongoing to address the concerns and offer technology-based treatments to increase gene therapy safety.

3. It might encourage gene doping

While gene doping doesn’t presently exist, it’s a procedure that might equalize educational opportunities, and athletics should equal technology access be allowed. If someone succeeds as a result of gene therapy when their success levels might not be the same without it, an ethical concern may arise, especially in athletic competitions.

4. Religious concerns may arise

A man’s genetic makeup manipulation is unacceptable for people with solid religious beliefs. They may consider altering genes as interfering with nature, amounting to questioning God’s will.

5. Its potentially dangerous

There’s a concern that harmful effects may occur when gene therapy gets to other body cells besides the faulty ones. If the reproductive cells are altered without detection or intention, they can be passed on to the patient’s offspring.


While gene therapy may be an effective treatment, it requires perfection for sure results and safety. Familiarize yourself with its pros and cons before trying to know what to expect.