What You Need to Know About Gene Therapy

Updated on December 5, 2023
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The last few decades have seen advancements in medicine and technology reaching unprecedented heights. Treatments for several lethal disorders have been discovered, making researchers dive deeper and deeper into the complexities of human biology. One such treatment is the use of gene therapy to cure diseases.  

Gene therapy is an advanced form of treatment that targets the genes in DNA responsible for causing certain diseases. Breakthroughs in genetics have the medical community excited about the possibilities gene therapy can unlock in the field of medicine. In this article, we’ll go through some of the basics of gene therapy.

How It Works

Gene therapy is a one-time treatment that aims to eliminate the root causes of certain disorders – faulty genes. The earliest methods used were gene transfer or gene addition. The former works by introducing a new gene to fight the disease, and the latter works by inserting a non-faulty gene to take the place of the misworking one. More recent developments in genomics have led to genome editing that modifies the malfunctioning portion of the existing DNA instead of introducing new genes into it. This is done through molecular tools like CRISPR-Cas9 and is being studied to turn genes on and off, along with fixing genetic alterations and removing portions of DNA that are causing impairments.   

Approved Gene Therapy Treatments

Gene therapy is still new, and it is mainly being used in research and clinical trial settings. A few of the simplest, most common gene therapy treatments approved by the FDA for use in the US are: 

  • Luxturna: A treatment procedure for a rare form of inherited vision loss – retinal dystrophy – involving a subretinal injection 
  •  Zolgensma: A treatment for spinal muscular atrophy, SMA 
  • ABECMA: A treatment for Multiple Myeloma in adults only to be used on treatment-resistant patients or those who have relapsed even after four types of therapies 

You can find a list of all FDA-approved treatments here. Hundreds of potential treatments are currently undergoing trials to treat genetic conditions that range from cancers to HIV/AIDS. 

Safety Concerns

The treatments that are approved pose minimum risk to the patient. Trials are usually carried out on other living organisms and are only used in clinical and research settings on humans after their safety is assured as much as possible by researchers. The complications that are looked out for include:

  • Rejection by the patient’s immune system
  • Other cells becoming unintended targets 
  • Viral infections
  • Tumors

It is to be noted that these gene treatments will only affect the individual being treated and not impact the chances of the previous disease being transmitted to their offspring. Offspring are only affected if the germ cells – reproductive cells – are modified, and such therapies are not approved yet. 


Gene therapy is a field that shows tremendous potential for growth; it can be expected to make considerable contributions to the field of medicine. Pediatric intervention to correct genetic abnormalities before they cause significant damage can especially help improve the quality of life at later stages. 

Ignorance often causes individuals to perceive it as threatening though safety concerns are almost non-existent in approved therapies. If you or someone you know is suffering from a disorder being researched for gene therapy and you wish to participate, consult your doctor. You can find a database of all trials being conducted at clinicaltrials.gov. Note that some people may have specific antibodies and won’t be able to participate in the trials as of yet. However, research is being conducted to step around this situation to make gene therapy possible for everyone.

The Editorial Team at Healthcare Business Today is made up of skilled healthcare writers and experts, led by our managing editor, Daniel Casciato, who has over 25 years of experience in healthcare writing. Since 1998, we have produced compelling and informative content for numerous publications, establishing ourselves as a trusted resource for health and wellness information. We offer readers access to fresh health, medicine, science, and technology developments and the latest in patient news, emphasizing how these developments affect our lives.