Breaking News
December 11, 2017 - MIT researchers discover new way to make bacteria vulnerable to existing antibiotics
December 11, 2017 - Office Workers Don’t Like Being Chained to Their Desks: MedlinePlus Health News
December 11, 2017 - Study reveals how a very low calorie diet can reverse type 2 diabetes
December 11, 2017 - University of Adelaide receives $23.2 million for preterm birth research
December 11, 2017 - Tough Flu Season Ahead: Vaccine May Only Be 10% Effective
December 11, 2017 - Getting Self-Driving Cars on the Road Soon Might Save Lives: MedlinePlus Health News
December 11, 2017 - Enterovirus vaccine prevents virus-induced diabetes in a T1D experimental model
December 11, 2017 - Solar retinopathy or sun damage to the eyes – a case report
December 11, 2017 - Cancer risk with birth control pills emerges again in latest study
December 10, 2017 - Deer Hunters: Put Safety First: MedlinePlus Health News
December 10, 2017 - High blood pressure is redefined as 130, not 140: US guidelines (Update)
December 10, 2017 - Unusual neuroinflammation may underlie cognitive problems in cART-treated HIV patients
December 10, 2017 - Racial differences in parents’ reports of child’s autism symptoms may contribute to delayed diagnosis
December 10, 2017 - Taurine could boost effectiveness of existing multiple sclerosis therapies
December 10, 2017 - The man with a young woman’s heart
December 10, 2017 - Patient bedside remains important component of medical education for students
December 10, 2017 - Nutrition labeling has little impact on sodium consumption
December 10, 2017 - ‘Obesity paradox’ not present among people with new cases of cardiovascular disease
December 10, 2017 - SLU scientists provide promising approach in designing new drugs for DMD
December 10, 2017 - Can PD-1 Inhibitor Help Eradicate HIV?
December 10, 2017 - Checking Prices for Medical Procedures Online? Good Luck: MedlinePlus Health News
December 10, 2017 - Researchers find bacteria tied to esophageal cancer
December 10, 2017 - Boy’s Double Hand Transplant Changed His Brain
December 10, 2017 - Memo to Doctors: Spit Out the Bad News: MedlinePlus Health News
December 10, 2017 - Two years of extended anastrozole therapy proved as effective as five years in clinical trial
December 10, 2017 - Using digital devices before bed may contribute to sleep and nutrition problems in children
December 10, 2017 - Are You Sure That’s What the Doctor Said About Your Leukemia?: MedlinePlus Health News
December 10, 2017 - Low vitamin D levels at birth linked to higher autism risk
December 10, 2017 - Alcohol use in movies influences onset of drinking among 10- to 15-year-olds
December 10, 2017 - For children with ADHD, a brief, school-based program can help dramatically with homework problems, study finds
December 10, 2017 - Surgical Residents Prime Candidates for Stress, Depression, Alcohol Abuse: MedlinePlus Health News
December 10, 2017 - Young athletes commonly develop bone marrow edema in the joints of the lower spine
December 10, 2017 - Coordinated approach in emergency care of heart attacks saves lives
December 10, 2017 - Patients OK With Fewer Opioids After Gallbladder Surgery
December 10, 2017 - Obamacare May Have Helped More Americans Quit Smoking: MedlinePlus Health News
December 10, 2017 - Long-term opioid use does not increase risk of Alzheimer ‘s disease
December 10, 2017 - Brief test can help determine whether drugs, talk therapy work better for treating anxiety
December 10, 2017 - Bioelectronic ‘nose’ can easily sniff foul smells in rotting food
December 10, 2017 - Spontaneous humor can be helpful for cancer patients to deal with distress
December 10, 2017 - Study finds mechanism of resistance to checkpoint inhibitors and way to reverse it
December 10, 2017 - ‘Lesser of two evils’ argument used to defend antipsychotic overuse for dementia
December 10, 2017 - Physical activity in middle age linked to lower incidence of joint symptoms
December 10, 2017 - Optogenetics research sheds new light on social-spatial learning
December 9, 2017 - Guidelines for MRI breast cancer screening not followed in community settings
December 9, 2017 - New first line combination therapy shows promising results in patients with advanced NSCLC
December 9, 2017 - Catheter-Directed Lysis Adds Risk, Not Benefit, for Proximal DVT
December 9, 2017 - Alzheimer’s Cases to Double by 2060: Report: MedlinePlus Health News
December 9, 2017 - New care model closes significant gap in addiction treatment
December 9, 2017 - PARP inhibitor can be new treatment option for patients with metastatic breast cancer, BRCA mutations
December 9, 2017 - Study shows how electrical stimulation in the brain instructs appropriate response
December 9, 2017 - Researchers develop molecular beacon to find hypoxic spots in real time
December 9, 2017 - Many Hospice Workers Lack Their Own End-of-Life Directives: MedlinePlus Health News
December 9, 2017 - New compound stops progressive kidney disease in its tracks
December 9, 2017 - Study: Changes in the brain after childhood limb loss can be reversed
December 9, 2017 - Semaglutide (Ozempic) Approved for Type 2 Diabetes
December 9, 2017 - Study Untangles Disparity in Colon Cancer Survival Rates: MedlinePlus Health News
December 9, 2017 - Lymph node surgery may raise risk of arm morbidity in younger women
December 9, 2017 - Study reveals connection between restless sleep and Parkinson’s disease
December 9, 2017 - Modern non-invasive diagnostics can get clear picture of the heart’s condition
December 9, 2017 - New method helps measure effect of one brain region on another in depression
December 9, 2017 - Mount Sinai study uses adaptive optics to examine retinal eye damage from solar eclipse
December 9, 2017 - Scientists introduce polyproline as effective cryoprotectant for monolayers of cells
December 9, 2017 - Simple molecular test may help predict presence of high-grade pancreatic cancers
December 9, 2017 - High levels of burnout, stress for U.S. surgical residents
December 9, 2017 - Parental lifespan genes could pave way for new therapeutic targets to prolong life
December 9, 2017 - About Half of Americans Get Health Care in ER: MedlinePlus Health News
December 9, 2017 - People with epilepsy may gain from common sleep apnea treatment
December 9, 2017 - Anti-impotence drug found to be ineffective at improving outcomes for IUGR pregnancies
December 9, 2017 - High risk of burnout, depression, and low quality of life found among health care workers
December 9, 2017 - Higher educational attainment associated with lower risk of Alzheimer’s disease
December 9, 2017 - Researchers seek to understand role of novel protein in tumor metastasis
December 9, 2017 - Research brings scientists one step closer to understanding development of ALS
December 9, 2017 - Women and Caregiving: Facts and Figures
December 9, 2017 - Mindfulness meditation found to relieve the stress of waiting for bad news
December 9, 2017 - Common fungus has significant impact on mosquitoes’ ability to transmit dengue virus
December 9, 2017 - Short online program improves ability of teen girls to communicate about safe sex
December 9, 2017 - Researcher discovers enzyme pathway involved in ALS pathogenesis
December 9, 2017 - Fusobacterium may play role in colon cancer growth, study finds
December 9, 2017 - Are Women Naturally Fitter Than Men?
December 9, 2017 - Celecoxib Strikes Out on Cancer Progression
Genetic treatment for blindness may soon be reality

Genetic treatment for blindness may soon be reality

image_pdfDownload PDFimage_print
Credit: CC0 Public Domain

Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting of the American Academy of Ophthalmology.

Patients in the study had a condition called Leber congenital amaurosis (LCA), which begins in infancy and progresses slowly, eventually causing complete blindness. This new, first-of-its-kind gene therapy is currently under review by the U.S. Food and Drug Administration for potential approval this year. There are currently no treatments available for inherited retinal diseases.

Ophthalmologist Stephen R. Russell, M.D., of the University of Iowa, is one of the lead researchers for this pioneering treatment. Data from the first randomized, controlled, phase 3 study showed that 27 of 29 treated patients (93 percent) experienced meaningful improvements in their vision, enough that they could navigate a maze in low to moderate light. They also showed improvement in light sensitivity and peripheral vision, which are two visual deficits these patients experience.

Approval could open the door for other gene therapies that could eventually treat the more than 225 genetic mutations known to cause blindness. It could be applied to retinitis pigmentosa, another inherited retinal disease caused by a defective gene. Or in the future, gene therapy could possibly provide key proteins needed to restore vision in more common diseases such as age-related macular degeneration.

LCA is rare, affecting about 1 in 80,000 individuals. It can be caused by one or more of 19 different genes. The treatment, called voretigene neparvovec (Luxturna, Spark Therapeutics), involves a genetically modified version of a harmless virus. The virus is modified to carry a healthy version of the gene into the retina. Doctors inject billions of modified viruses into both of a patient’s eyes.

Treatment doesn’t restore normal vision. It does, however, allow patients to see shapes and light, allowing them to get around without a cane or a guide dog. It is unclear how long the treatment will last, but so far, most patients have maintained their vision for two years.

More than 200 patients with LCA have participated in gene therapy trials since 2007. However, no gene therapy has gotten this close to FDA approval for retinal disease or any other eye disease. In October, an advisory committee to the FDA unanimously endorsed the treatment. The FDA isn’t obligated to follow the recommendations of its advisory committees, but it usually does. The agency is expected to make its decision no later than January 2018.


Explore further:
FDA panel mulls gene therapy for kids with rare eye disease (Update)

Provided by:
American Academy of Ophthalmology

Tagged with:

About author

Related Articles