According to the National Institutes of Health (NIH), approximately 7,000 rare diseases affect under 200,000 Americans each year. With such diversity, there are many different treatment plans that vary substantially from situation to situation.
Gene therapy is a relatively new treatment option for patients. Though this method is relatively new and considered experimental in medicine, it has proven to be immensely helpful in the right situation. Currently, the safety and efficacy of many gene therapy treatments are still under review. However, new findings may provide key information that can advance gene therapy for wider use.
What Is Gene Therapy?
Gene therapy involves a complex process of inserting desirable genes into a patient’s cells, often in addition to medication or surgical techniques. By providing a healthy copy of a particular gene that isn’t performing as it should, gene therapy hopes to reprogram a cell’s function and help it perform correctly.
There are currently three major approaches to gene therapy undergoing testing in the scientific community. These include:
- Inactivating mutated genes that are not performing their necessary functions
- Replacing a mutated gene with a healthy copy of that same gene
- Injecting a new gene into the patient’s body
The method used depends on the patient, the medical condition under treatment, and the options available for both.
How Can AAV Capsids Help?
Researchers have found that adeno-associated viral (AAV) capsids may be helpful to improve gene therapies. AAV are very simplistic viruses that naturally occur in the human body. They have a single strand of DNA, and though they are a virus, they do not cause symptoms or illnesses.
AAV capsids can act as a delivery system for gene therapies. Scientists can replace the virus DNA in the AAV capsids with new DNA that can benefit the patient. Because AAV is simple and can replicate without outside help (unlike other viruses), it can effectively deliver healthy DNA and genes to organs and tissues that are diseased or affected.
The Importance of High Throughput Screening
As a part of this therapy, researchers must select ideal AAV capsids in order to proliferate healthy DNA. With high throughput screening, researchers are able to utilize automated technology to easily vet entire libraries of AAV capsids for options that can help achieve their desired outcome. During this round of selections, researchers look for an AAV that has a high rate of infection. This way, the new DNA will be replicated more effectively.
High throughput screening is important because it allows researchers to quickly and effectively find AAV capsids that will be effective, rather than individually testing each option in the lab. High throughput screening also gives scientists a view of the AAV’s full set of characteristics, providing more necessary information about potential treatment use cases.
Future Implications
The combination of AAV use and high throughput screening systems has the capacity to make gene therapy more reliable. If researchers have the ability to vet AAV capsids before they are used, they can exert better control over gene therapy treatments with improved outcomes.
Currently, gene therapy is considered a relatively experimental option. However, with the precision and outcomes that high throughput screening provides, gene therapy may become a more effective, widely used treatment for serious diseases of many kinds.
Sources:
https://www.news-medical.net/news/20200318/High-throughput-screening-of-AAV-vector-capsid-libraries-reveals-unexpected-insights.aspx (From March 2020)
https://medlineplus.gov/genetics/understanding/therapy/genetherapy/ (Last updated May 2021)
https://www.askbio.com/aav-gene-therapy/
https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
