Improving Sight Through Gene Therapy Research Studies
Gene therapy is a newer, innovative technology that is being researched as an effective treatment option for many medical conditions, including those affecting the retina and vision loss.
At Austin Retina Associates, we are dedicated to advancing treatments that can help improve eyesight and restore vision loss through participation in leading-edge clinical trials. For decades, our retinal specialists have offered patients the opportunity to receive novel procedures and therapies through enrollment in clinical research studies.
Vitreoretinal surgeon Dr. Edward Wood frequently serves as a principal investigator in retina research trials and is sharing his expertise on gene therapy to treat retinal conditions.
How does gene therapy work?
Gene therapy is a medical technology that treats disease by altering genes or gene expression in a certain part of the body, in our case, the retina.
Gene therapy involves packaging a specific DNA sequence of a target gene into a delivery vector which is often an inactivated virus. Once delivered into the cell, the gene is expressed, generating protein(s) to restore cellular function and address the underlying disease process.
Common ophthalmic gene therapy indications include inherited (genetic) retinal disease, but more common diseases (such as age-related macular degeneration (AMD) and diabetic retinopathy (DR)) may also be targeted with approaches such as gene therapy. When targeting more common retinal diseases, the gene encoding a therapeutic drug is delivered into the retina, allowing the retina to become its own bio-factory and make the therapeutic drug on its own.
How is gene therapy delivered?
In the field of retina care, there are many different ways to administer gene therapy into the eye: (1) in office intravitreal injections, (2) in office suprachoroidal injections, and (3) sub-retinal gene therapy which is administered under the retina during vitrectomy surgery.
What are the benefits of gene therapy to the patient?
When targeting more common retinal diseases such as macular degeneration (AMD), allowing the retina to make the therapeutic drug on its own has the potential for reduced need for in-office visits/treatments and possibility more disease stability.
What else should patients know before considering gene therapy?
Because gene therapy modifies or supplements the DNA of your body, it is not a treatment that can be easily reversed or removed, and there are rare risks including inflammation. Gene therapy is typically a one-time treatment regimen, but some patients may still require additional treatment on top of the gene therapy.
Austin Retina Associates Patients Benefit from Clinical Research Trials
In addition to the hard work put in by our team of retinal specialists who seek and identify promising trials that could help improve retinal health and vision, we are especially grateful to our patients who are eager to participate in them.
One Austin Retina patient who has been a part of multiple clinical research trials is 76-year-old Marilys Leslie, a retired teacher.
In 2020, Marilys noticed problems with her vision when she would read music and the lines would mix together and some lines would disappear in the middle of her sight. Having a family history of macular degeneration, Marilys quickly made an appointment and was diagnosed with the condition. She immediately signed up to participate in AMD research studies with the goal of helping others maintain their eyesight. AMD caused her mother to lose vision in one of her eyes because at that time, treatments were not as advanced as they are today.
Marilys’ vision has improved greatly following the trials allowing her to return to the things she has always loved like reading, needlepoint and playing piano.
Her advice to others is “as soon as you notice symptoms, be seen, and make sure you do your annual vision checks.” She further adds to be aware of symptoms of deteriorating retinal health and vision loss, especially as you age.
Consult With a Retina Specialist in Austin Today
To learn more about the current clinical research trials being offered at Austin Retina Associates, visit us here.