Gene therapy to treat blindness on the anvilAugust 1st, 2009 - 2:17 pm ICT by ANI
Washington, Aug 1 (ANI): In a novel study, American and Arab researchers are working together to develop a gene therapy that would help treat a rare, hereditary retinal disease.
The therapy has been shown to restore lost vision in animal models of retinitis pigmentosa (RP).
The RP patients first experience defective dark adaptation or “night blindness,” followed by reduction of the peripheral visual field known as tunnel vision, sometimes followed by loss of central vision late in the course of the disease.
The team led by Dr Kang Zhang, professor of ophthalmology at the University of California, San Diego School’s Shiley Eye Center and director of the UCSD Institute for Genomic Medicine, and Dr Fowzan Alkuraya, senior clinical scientist and head of developmental genetics unit at King Faisal Specialist Hospital and Research Centre, Saudi Arabia are using receptor protein called MERTK that is expressed in the retinal pigment epithelium,
It is the pigmented cell layer just outside the retina that closely interacts with photoreceptors in the maintenance of visual function.
Patients with loss of MERTK function have a defect in phagocytosis - a mechanism used to remove pathogens and cell debris.
As a result of this defect, debris accumulates between the photoreceptors and retinal pigment epithelium, resulting in death of photoreceptors and loss of vision.
The researchers plan to deliver the MERTK gene in a viral vector - a carrier commonly used to deliver genetic material to treat these cells in order to restore function of photoreceptors.
It showed that viral vector delivery of MERTK corrected the mutant gene and restored vision.
The same type of vector has been successfully tested in both animals and humans for a similar type of early-onset retinal degeneration called Leber’s congenital amaurosis.
The research team’s next step is to show that such gene therapy is safe in further animal studies, to be conducted in China, along with additional rat studies that will be conducted at UC San Diego and at the University of Florida. (ANI)
- Stem cell therapy for age-related macular degeneration may soon be a reality - Mar 25, 2011
- Night blindness cured in mice with special cells - Apr 19, 2012
- Promising results of gene therapy to treat eye diseases - Aug 14, 2010
- Sharks are colour blind, says study - Sep 20, 2012
- Stem cell therapy may help restore sight in retinitis pigmentosa sufferers - Feb 25, 2010
- Eye rich source of flexible adult stem cells - Jan 09, 2012
- New prosthetic device offers to hope to the visually impaired - Nov 19, 2010
- New hope for restoring vision in the blind - Sep 22, 2010
- Study sheds light on inherited retinal disease - Jan 18, 2010
- Breakthrough gene therapy could preserve vision - Aug 17, 2010
- Human embryonic stem cells offer new hope for people with retinal degeneration - Oct 02, 2009
- London hospital to do Europe's first embryonic stem cell trial - Sep 24, 2011
- Researchers isolate protective mechanism to save sight - Nov 17, 2009
- Eye cells deemed to be retinal stem cells are actually normal adult cells - Mar 31, 2009
- Compound likely to regenerate vision in humans - Jul 26, 2012
Tags: california san diego, cell debris, clinical scientist, congenital amaurosis, dark adaptation, developmental genetics, genomic medicine, kang zhang, king faisal specialist hospital, king faisal specialist hospital and research centre, night blindness, novel study, photoreceptors, pigment epithelium, receptor protein, retinal pigment, rp patients, shiley eye center, university of california san diego, viral vector