To use all functions of this page, please activate cookies in your browser.
With an accout for my.bionity.com you can always see everything at a glance – and you can configure your own website and individual newsletter.
- My watch list
- My saved searches
- My saved topics
- My newsletter
UF researchers develop gene therapy that could correct a common form of blindness
24-01-2012: A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. The findings are published in the Proceedings of the National Academy of Sciences online. Several complex and costly steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives.
“Imagine that you can’t see or can just barely see, and that could be changed to function at some levels so that you could read, navigate, maybe even drive — it would change your life considerably,” said study co-author William W. Hauswirth, Ph.D., the Rybaczki-Bullard professor of ophthalmology in the UF College of Medicine and a professor and eminent scholar in department of molecular genetics and microbiology and the UF Genetics Institute. “Providing the gene that’s missing is one of the ultimate ways of treating disease and restoring significant visual function.”
The researchers tackled a condition called X-linked retinitis pigmentosa, a genetic defect that is passed from mothers to sons. Girls carry the trait, but do not have the kind of vision loss seen among boys. About 100,000 people in the U.S. have a form of retinitis pigmentosa, which is characterized by initial loss of peripheral vision and night vision, which eventually progresses to tunnel vision, then blindness. In some cases, loss of sight coincides with the appearance of dark-colored areas on the usually orange-colored retina.
“That was a great advance, which showed that gene therapy is safe and lasts for years in humans, but this new study has the potential for a bigger impact, because it is treating a form of the disease that affects many more people,” said John G. Flannery, Ph.D., a professor of neurobiology at the University of California, Berkeley who is an expert in the design of viruses for delivering replacement genes. Flannery was not involved in the current study.
The X-linked form of retinitis pigmentosa addressed in the new study is the most common, and is caused by degeneration of light-sensitive cells in the eyes known as photoreceptor cells. It starts early in life, so though affected children are often born seeing, they gradually lose their vision.
“These children often go blind in the second decade of life, which is a very crucial period,” said co-author Alfred S. Lewin, Ph.D., a professor in the UF College of Medicine department of molecular genetics and microbiology and a member of the UF Genetics Institute. “This is a compelling reason to try to develop a therapy, because this disease hinders people’s ability to fully experience their world.”
- 1Pharma’s New Hero: Supergenerics Save Money and Improve Drugs
- 2Pro Bono Bio Launches Flexiseq: A Novel Approach to the Treatment of Osteoarthritis
- 3Rosetta Resolver® Gene Expression Data Analysis System licensed by Aventis Pharmaceuticals, Inc.
- 4Panspecific antibodies: Novimmune successful in multiple specificities
- 5Researchers divide enzyme to conquer genetic puzzle
- 6Arkema and CJ officially launch the construction of their Bio-Methionine and Thiochemicals complex in Malaysia
- 7Merck & Co., Inc. Opens Asia Pacific Regional Headquarters in Singapore
- 8Pharmexa A/S sells Pharmexa-Epimmune to the Korean company VaxOnco, Inc.
- 9MIV-210 - a potential drug against hepatitis B and HIV - enters phase II
- 10Bayer acquires exclusive rights to Bioton’s insulin SciLin
- Brain changes are associated with casual marijuana use
- Nanocrystalline cellulose modified into an efficient viral inhibitor
- Pharmacist-led interventions show high success rates for post-stroke care
- Targeting cancer with a triple threat
- Potent, puzzling and (now less) toxic: How antifungal drug works