Vision Loss Breakthrough: Is Gene Therapy the Future of Eye Health?

One of the main causes of vision impairment and blindness is the impact on the retina, the light-sensitive layer in the back of the eye, by various diseases. Some vision loss issues may be treated using gene therapy, and recent developments in research might soon change the therapeutic landscape for eye health, which will be a very satisfying outcome.   

By inserting a new gene or replacing a damaged gene, gene therapy aims to alter the behavior of the cells. For inherited retinal disorders (IRDs) like retinitis pigmentosa (RP) or Stargardt disease, which are brought on by a damaged or mutated gene, gene therapy is at the absolute forefront of novel medical approaches that are now being adopted by doctors. Other forms of vision loss, such as diabetic macular edema (DME), age-related macular degeneration (AMD), and corneal eye disease, are also being investigated with gene therapy, and promising results are produced. Keep reading to find out everything about the gene therapy being developed to cure vision loss.  

What is Gene Therapy? 

Gene therapy is a cutting-edge medical procedure that treats disease by using gene material, and the technology enables doctors to address specific problems by altering your genetic composition instead of employing conventional therapy like medicine and surgery. In this manner, medical professionals can treat the disease's root cause or instruct your body to create desired proteins in large quantities.  

Gene therapy involves introducing genetic material into your cells, and the way your cells make proteins is then altered by this genetic material. It can lower the amounts of certain proteins that cause sickness, boost the synthesis of functional proteins, and make new or altered proteins within a cell.  

How does Gene Therapy Work?  

The genetic material of your body is stored in chromosomes within the cell nucleus, and the DNA, which makes up each chromosome, contains information that determines your individual characteristics. Genes are specific regions of DNA, and protein synthesis instructions are provided by these genes. The way your body works is significantly influenced by proteins, and that is why, the way proteins function may be changed by a slight alteration to the DNA in your genes. 

As cells age or after being exposed to specific chemicals or environmental variables, gene variations, also known as genetic alterations, take place. In some cases, these genetic alterations are recognized and corrected by cells, but in other cases, they may result in a sickness or illness for which you will require medical attention. These gene variations may also be inherited from your biological parents, which might result in illness from a young age. Healthcare professionals want to treat the root cause of illness by employing gene therapy because your genes are your body's blueprint, and correct gene therapy can fix any mistakes or add missing pieces.  

Application of Gene Therapy for Vision Loss 

• Gene Editing 
  This therapy is gene-specific and requires knowledge of the gene mutation. Molecular scissors are employed in gene editing to "edit" a damaged gene by removing the mutation, and RP (retinitis pigmentosa) and Usher syndrome are among the IRDs (inherited retinal disorder) for which this type of gene therapy is being used as a treatment.  

• Gene Replacement
  In gene replacement, a damaged gene is replaced by a new one that is inserted into the cell. Luxturna, the first licensed gene therapy, is based on gene replacement, which is being employed for numerous IRDs.   

• Gene Therapy to Replace Anti-VEGF Injections
  Injections of anti-VEGF are the most widely used therapy for DME (diabetic macular edema) and AMD (age-related macular degeneration). Uncontrollably growing retinal blood vessels that leak fluid into the eye are the source of visual loss in both AMD and DME and anti-VEGF medications function by reducing blood vessel development. The possibility of introducing genes that direct retinal cells to make their own anti-VEGF is being tested in clinical studies in order to lessen the need for repeated injections.  

• Gene Therapy to Stop Cell Death
  When retinal cells that aid in the perception and transmission of light signals die, vision loss happens, so researchers are creating "neuroprotective" gene treatments, which insert genes into retinal cells to prevent their death. Since this kind of gene therapy is not gene-specific, it can be utilized for disorders like AMD that may not have a genetic origin, IRDs with several mutation types, or IRDs for which the gene mutation has not yet been found. 

What are the Most Serious Vision Loss Reasons?   

• Retinitis pigmentosa - The retina is the area of the eye that contains unique cells that respond to light, and when you have Retinitis pigmentosa, the retinal cells degrade as the disease worsens. This first causes night vision issues, which are then followed by peripheral vision loss in the patient. 
• Retinopathy of prematurity - Some preterm newborns get this retinal disease when blood vessels proliferate in the wrong areas of their eye. Their retina may be damaged by scar tissue, which might result in blindness and severe vision loss. 
• Age-related macular degeneration - The macula, the area of the retina that regulates central vision, is impacted by this disorder, and reading and facial recognition becomes extremely challenging when significant central vision loss occurs. 
• Leber hereditary optic neuropathy - This term describes a kind of progressive eyesight loss that is inherited, and males are more affected than females by this disease for unclear reasons. 
• Diabetes-related retinopathy - If you have diabetes and your eye's blood vessels are compromised, you may get this disorder. As a result, blindness may develop if the visual loss worsens over time or if treatment is not received on time. 
• Glaucoma - You receive damage to your optic nerve when you have this disease, and in more severe stages of the illness, blindness may result from visual loss.  

Challenges of Gene Therapy for Vision Loss  

Numerous eye conditions may be treated using gene therapy, which has shown great promise, but for those seeking this treatment, there are a few obstacles and crucial factors to consider. The most promising gene treatments at the moment are those that target specific genes and IRDs, such as RP or (LCA) Leber congenital amaurosis, however, keep in mind that making a gene treatment for every gene or mutation may be very costly and time-consuming.  

Furthermore, this kind of therapy is only effective for those who have the precise gene mutation for which it is intended. The disease's stage presents another difficulty because gene therapy techniques will be effective only when there are sufficient healthy retinal cells left. That is why, other strategies, such as stem cell therapy or artificial retinas, could be more suitable when there aren't many healthy retinal cells left in the eyes.  

Final Remarks  

Gene therapy for vision loss has opened a new frontier that so many people will greatly benefit from! Treatment options for inherited and acquired ocular diseases have expanded because of gene therapy, and doctors anticipate seeing a larger practical use of this ground-breaking technology as research progresses.  

If you are considering gene therapy for vision loss, contact Pathkind Labs to get the necessary tests done by experienced professionals!