Luxturna, Gene Therapy and Your Inherited Retinal Disease

Caption: Scene as Viewed by Person with Retinitis Pigmentosa In December 2017, news broke to great fanfare that the FDA had approved the first ever gene therapy for a genetic disease. Luxturna (voretigene neparvovec-rzyl) had been proven to restore vision in people living with inherited retinal diseases. Stories about children seeing their parents’ faces for … Continued

The Reality and Costs of Gene Therapy for Eye Disease: Who Will (or Can) Pay?

As our readers know, VisionAware is committed to providing current and reliable information about eye and vision research that is relevant to adults and older adults, many of whom experience late-life vision loss resulting in low vision. During the past several years, gene therapy has gained traction and offers promise for treating (but not yet … Continued

Treating Macular Degeneration with Gene Therapy: New Research Shows Promise but Also Has Limitations

Currently, there are a number of treatments for wet age-related macular degeneration, including the drugs Lucentis, Eylea, and Avastin, administered by injection with a very small needle directly into the eye after the surface has been numbed (also called “intra-vitreous injection”). There are also a number of treatments that have proven to be inconclusive or … Continued

New Genetic Research in Diabetes Identifies a Protein That May Stop or Reduce Abnormal Blood Vessel Growth in the Retina

New genetic research in diabetes, led by a team from Harvard Medical School, has identified a potential new therapy targeting RUNX1 (explained below) that significantly reduced abnormal blood vessel growth in the retina, which is a hallmark of advanced diabetic eye disease. Although the research has been conducted only with “in vitro” (explained below) laboratory … Continued

New Research: Gene Editing as a Potential Treatment for Wet Macular Degeneration

At present, there are a number of current treatments for wet age-related macular degeneration, including the drugs Lucentis, Eylea, and Avastin, administered by injection with a very small needle directly into the eye after the surface has been numbed. There have also been a number of treatments that have proven to be inconclusive or unsuccessful … Continued

Meet Author Irv Arons and Learn More About Gene Therapy and Gene Editing for Eye Disease

Irv Arons is the creator of – and driving force behind – Irv Arons’ Journal, an online compendium of the latest information on ophthalmics, medical lasers, clinical trials, developments in stem cell therapy and gene therapy, and emerging drug therapies and treatments for retinal diseases, including age-related macular degeneration. Until his retirement in 2005, Irv … Continued

New Genetic Research in Macular Degeneration: The International AMD Genomics Consortium

The International Age-Related Macular Degeneration (AMD) Genomics Consortium, which includes 26 centers worldwide, has published new data and information about the role of genes and their contribution to the risk of macular degeneration. Previously, researchers had identified 21 regions of the human genome – called loci – that are associated with an increased risk of … Continued

New Research: Gene Therapy Restores Some Vision in Clinical Trials for Leber Congenital Amaurosis (LCA)

Gene therapy for the treatment of specific eye and retinal disorders, such as Leber congenital amaurosis (LCA), has shown promise, but research has produced uneven results thus far and has not produced a cure. To date, gene therapy studies have raised concerns among researchers, including inconsistent initial and longer-term results, such as this LCA gene … Continued

Is Glaucoma a Genetic Disease? New and Innovative Genetic Research Shows Promise in the Treatment of Glaucoma

New glaucoma research from the United States and China indicates that a genetic interaction may prove to be a key component in the development and progression of open-angle glaucoma. Although this genetic research has been conducted only with laboratory mice, the concept shows great promise for developing and identifying effective therapies for treating – and … Continued

Optogenetics: Can This Innovative Gene Therapy Treat Degenerative Retinal Disease and Possibly Restore Sight?

A research group of Swiss and German scientists has restored vision to mice with a condition similar to retinitis pigmentosa (RP) by introducing engineered light-sensing proteins into their eyes, via a process known as optogenetics. Optogenetics is a still-experimental treatment for a variety of blinding retinal disorders that uses gene therapy to enable retinal and … Continued