Navigation Links
Gene therapy for blindness clears hurdle in mice

University of Florida researchers have used an experimental therapy in mice to shut down a gene that plays a crucial role in a leading cause of inherited blindness.

The technique, detailed in an upcoming issue of Vision Research, involves injecting the eye with a bit of genetic material called interfering RNA, which helps disable the gene.

Normally the gene is essential for healthy eyesight, but mutated versions of it are passed from generation to generation in some families and can lead to blindness.

Disabling the gene is a step toward developing a gene therapy to treat people with retinitis pigmentosa, an inherited disease that attacks the light-sensing cells in the eye. It affects about one in 60,000 people, with an estimated 1.5 million people afflicted worldwide.

"One of the causes of the disease is mutated gene expression," said Marina Gorbatyuk, Ph.D., an assistant professor of molecular genetics and microbiology in the UF College of Medicine. "We work with rhodopsin, which is the main retinal protein. Without it, or if it is mutated, people simply won’t see."

Mutated forms of the rhodopsin produce a toxic protein in the retina that kills cells that receive light. People with the disease usually notice symptoms between the ages of 10 and 30. At first they have problems seeing in dimly lit places, followed by loss of their peripheral sight. The rate of progression varies, but most patients are blind by 40.

UF Genetics Institute researchers engineered the interfering RNA into a virus, which in turn was injected below the retinas in more than a dozen normal mice. Analysis showed the technique reduced the amount of rhodopsin by about 60 percent.

With the gene drastically muzzled, scientists have begun experiments to create a therapy in which healthy versions of the gene can be introduced into the eye using an apparently harmless virus to deliver the genetic material.

"If we reduce the am ount of protein formed by mutated rhodopsin, that may be sufficient to maintain vision in people who are affected by retinitis pigmentosa," Gorbatyuk said. "The second step, introducing the normal gene to the retina, will show whether we are able to restore vision in this model or not."

If both steps are perfected, scientists plan to study the treatment in a larger animal model and then possibly move to a human clinical trial.
'"/>

Source:University of Florida


Related biology news :

1. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
2. Columbia research lifts major hurdle to gene therapy for cancer
3. Combination therapy boosts effectiveness of telomere-directed cancer cell death
4. Gene therapy converts dead bone graft to new, living tissue
5. Study identifies predictors of HIV drug resistance in patients beginning triple therapy
6. New imaging method gives early indication if brain cancer therapy is effective, U-M study shows
7. Muscle-targeted gene therapy reverses rare muscular dystrophy in mice
8. New therapy for HIV/AIDS eliminates needles and excessive toxicity
9. New Treatment Rivals Chemotherapy For Lymphoma, Study Finds
10. Gene therapy for Parkinsons disease moves forward in animals
11. Pulsating ultrasound enhances gene therapy for tumors

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:1/18/2016)... , Jan. 18, 2016  Extenua Inc., ... that simplifies the use and access of ubiquitous ... go-to-market partnership with American Cyber.  ... extensive experience leading transformational C4ISR and Cyber initiatives ... integrating the latest proven technology solutions," said ...
(Date:1/13/2016)... January 13, 2016 --> ... a new market report titled - Biometric Sensors Market - ... 2015 - 2023. According to the report, the global biometric sensors ... anticipated to reach US$1,625.8 mn by 2023, expanding at ... terms of volume, the biometric sensors market is expected ...
(Date:1/11/2016)... , Jan. 11, 2016 Synaptics Incorporated ... interface solutions, today announced that its ClearPad ® ... (TDDI) products won two separate categories in the 8 ... Innovator and Best Technology Breakthrough. The Synaptics ® ... a simplified supply chain, thinner devices, brighter displays and ...
Breaking Biology News(10 mins):
(Date:2/4/2016)... SHENZHEN, China , Feb. 4, 2016 ... government, and various medical institutions attended a ceremony in ... provide integrative, personalized cell therapy in 2016. ... the "Shenzhen Clinical Translation Platform for Personalized Cell Therapy" ... Shenzhen Regional Cell Production Center, both subsidiaries of Beike ...
(Date:2/4/2016)...  Spherix Incorporated (Nasdaq: SPEX ) -- an intellectual property ... intellectual property, today provided an update on the Company,s ... of Texas and announcing that ... Partes Re-examination ("IPR") proceedings that VTech and Uniden ... initiated on only certain claims of two of the ...
(Date:2/3/2016)... DIEGO , Feb. 3, 2016   ... company with the first pluripotent stem cell-derived islet ... diabetes in clinical-stage development, today announced that ViaCyte ... Pharmaceutical Companies of Johnson & Johnson, have agreed ... group into ViaCyte.  The agreement provides ViaCyte with ...
(Date:2/3/2016)... BRUNSWICK, N.J. , Feb. 3, 2016 /PRNewswire-USNewswire/ ... grants totaling more than $1 million for researchers ... are working on health-related research that demonstrates exciting ... this round of funding for the New Jersey ... for faculty members at these educational institutions— Princeton ...
Breaking Biology Technology: