Browse By Topic: Gene Therapy

Ongoing research in genetics, optogenetics, and gene therapy for a wide range of eye diseases and disorders.

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 the first time and adults putting away their white canes forever were widely circulated. As a vision rehabilitation specialist, I’ve been approached by clients who ask how they too can become pioneers in the nascent field of gene therapy. It was time to look into


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 curing) a range of eye diseases, including retinitis pigmentosa,


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

The Lancet logo 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


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

A retina with diabeticretinopathy 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


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 after undergoing clinical trials, including


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 was a consultant to the ophthalmic industry for over 30 years, and to the medical laser industry for over 20 years.


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 AMD. The new research, published in Nature Genetics, increases the number of loci to 34.


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 therapy study from the National Eye Institute, which reported that


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 even preventing – glaucoma. Molecular Cell: the Research The research, entitled P16INK4a [a type of gene] Upregulation [increased


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 brain cells to respond to light. According to the researchers, "… optogenetic gene therapy, which selectively introduces genes encoding light-sensitive proteins into surviving retinal cells to act as


New Genetic Research Investigates the Link between Inflammation and Macular Degeneration

European researchers investigating the link between inflammation and age-related macular degeneration (AMD) have identified a protein, called FHL-1, that functions as a type of "regulator" to protect the eye from an attack by the immune system. According to lead author Dr. Simon Clark, this important genetic research has identified a new target for therapeutic drugs that can "reset" the immune imbalance in the eye, thus possibly preventing, or delaying the progression of, AMD. From the Journal of


Is Glaucoma a Genetic Disease? Three New Research Projects Pinpoint Six Specific Genes

Three new research projects exploring the role of genes – and six genes in particular – as possible causes of glaucoma have been published simultaneously in the August 31, 2014 online edition of Nature Genetics. Nature Publishing Group (NPG) is a publisher of scientific and medical information in print and online. NPG publishes a range of journals across the life, physical, chemical, and applied sciences and clinical medicine. Although research scientists are the primary audience, news summaries and articles make many of


Can Gene Therapy Provide a Cure for Retinal Disease? An Early-Stage Clinical Trial Says "Maybe"

A group of international researchers from the United Kingdom, the United States, the Netherlands, and Portugal have used gene therapy in a clinical trial to treat a small group of male subjects with choroideremia, a rare degenerative eye disease. This early-stage clinical trial demonstrates potential for the use of gene therapy to treat a number of additional retinal diseases, including age-related macular degeneration. The research, entitled Retinal gene therapy in patients with choroideremia: initial


Could the Loss of an Anti-Aging Gene Contribute to Wet and Dry Macular Degeneration?

A team of researchers has determined that the loss of a particular anti-aging, or aging-suppressor, gene – known as Klotho protein (KL) – induces retinal deterioration in mice and may contribute to both wet and dry age-related macular degeneration (AMD). The KL gene helps to protect against oxidative stress, which causes dry AMD, and inhibits harmful blood vessel growth in the eye, which is the primary cause of


Gene Therapy and Leber Congenital Amaurosis: Update from the National Eye Institute

The following update addressing the role of gene therapy in the treatment of Leber congenital amaurosis (LCA), an inherited and progressive eye disease, is adapted, with permission, from a National Eye Institute news brief entitled New Findings Suggest Need for Combined Strategy in Treatment of Rare Form of Blindness. The mission of the National Eye Institute is to "conduct and support research, training, health information dissemination, and other programs with respect to blinding eye diseases,


A Nationwide Study Is Investigating Genetic and Other Risk Factors for Macular Degeneration

Researchers at the Jules Stein Eye Institute of the University of California at Los Angeles are continuing to recruit participants for a nationwide study investigating the heredity and exposure risk factors that lead to the development of age-related macular degeneration. The GARM II Study The goal of the Genetics of Age-Related Maculopathy (GARM II) Study is:


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