Corporate Banner
Satellite Banner
Technology
Networks
Scientific Communities
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Improving Gene Therapy for Eye Diseases

Published: Friday, July 05, 2013
Last Updated: Friday, July 05, 2013
Bookmark and Share
Researchers developed a less invasive technique that delivers genes across the retinas of mouse and monkey eyes.

With further development, the method might be used in people to treat inherited diseases that cause the retina to degenerate and impair sight.

The retina is the light-sensitive tissue at the back of the eye. It converts light into electrical impulses that are sent to the brain through the optic nerve, allowing us to see.

Certain inherited diseases, such as Leber congenital amaurosis (LCA), cause the retina to degenerate and lead to blindness. Scientists have made progress using gene therapy to treat these eye diseases, and several clinical trials are underway. However, the current therapies require inserting a needle through the retina and injecting the engineered virus behind the retina. The procedure can disrupt fragile diseased retinas and delivers the therapy to only a limited region.

A research team led by Drs. John G. Flannery and David V. Schaffer at the University of California, Berkeley, set out to develop an improved approach. The virus typically used for gene therapy in the eye is adeno-associated virus (AAV). This harmless virus can’t normally get through the layers of retinal cells to reach affected photoreceptor cells when injected into the vitreous humor—the more easily accessible gel-like fluid at the center of the eye. The scientists devised an approach to engineer variants of the virus that could get through the retina's layers. They described their work, which was funded in part by NIH’s National Eye Institute (NEI) and the NIH Common Fund, on June 12, 2013, in Science Translational Medicine.

The researchers injected millions of genetically engineered variations of AAVs into the vitreous humor of transgenic mice and selected the variants that reached photoreceptor cells in the retina. After 3 rounds of selection, the scientists identified dozens of AAV variants capable of moving from the vitreous humor into the retina.

The most effective modified AAV, called 7m8, was used for further study. When injected into the vitreous humor of adult mice, 7m8 delivered genes throughout the retina and the optic nerve, but not into surrounding tissues outside of the eye. Using a more specific gene promoter restricted gene expression to photoreceptor cells.

The team tested 7m8 in mouse models of 2 inherited diseases in which mutated genes lead to retinal degeneration. In X-linked retinoschisis (XLRS), mutations in the gene encoding the retinoschisin protein cause splitting of the retina. LCA type 2 is caused by mutations in the RPE65 gene. Injections of 7m8 carrying functional copies of these genes into the vitreous humor improved vision in both mouse models.

The new technique also successfully delivered genes across the retina of adult macaques. The team is now planning to perform additional research to prepare the 7m8 AAV for study in humans. They hope to soon head into early clinical trials.

“Building upon 14 years of research,” Schaffer says, “we have now created a virus that you can inject into the liquid vitreous humor inside the eye and it delivers genes to a very difficult-to-reach population of delicate cells in a way that is surgically much less invasive and safer.”


Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,500+ scientific posters on ePosters
  • More Than 3,700+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

High-Resolution 3D Images Reveal the Muscle Mitochondrial Power Grid
NIH mouse study overturns scientific ideas on energy distribution in muscle.
Friday, July 31, 2015
Vital Protein in Healthy Fertilization Process Identified
Researchers at the National Institutes of Health have discovered a protein that plays a vital role in healthy egg-sperm union in mice.
Monday, July 27, 2015
Young South African Women can Adhere to Daily PrEP Regimen as HIV Prevention
NIH-funded study finds men in Bangkok, Harlem also successful in taking daily dose.
Saturday, July 25, 2015
Study Shows Promise of Precision Medicine for Most Common Type of Lymphoma
The study appeared online July 20, 2015, in Nature Medicine.
Tuesday, July 21, 2015
NIH Joins Public-Private Partnership to Fund Research on Autism Biomarkers
Biomarkers Consortium project to improve tools for measuring and treating social impairment in children with autism.
Tuesday, July 21, 2015
NIH Study Identifies Gene Variant Linked to Compulsive Drinking
Mice carrying the Met68BDNF gene variant would consume excessive amounts of alcohol.
Tuesday, July 21, 2015
HIV Control Through Treatment Durably Prevents Heterosexual Transmission of Virus
NIH-funded trial proves suppressive antiretroviral therapy for HIV-infected people effective in protecting uninfected partners.
Tuesday, July 21, 2015
Early Antiretroviral Therapy Prevents Non-AIDS Outcomes in HIV-infected People
NIH-supported findings illustrate manifold benefit of therapy.
Tuesday, July 21, 2015
Futuristic Brain Probe Allows for Wireless Control of Neurons
NIH-funded scientists developed an ultra-thin, minimally invasive device for controlling brain cells with drugs and light.
Saturday, July 18, 2015
House Votes in Favor of Bill Boosting NIH Funding
The US House of Representatives today overwhelmingly voted in favor of a bill that would increase funding to the NIH by about $10 billion, help speed the development of new drugs, and advance precision medicine initiatives.
Monday, July 13, 2015
NIH-funded Vaccine for West Nile Virus Enters Human Clinical Trials
Enrollment is expected to be completed by December 2015.
Tuesday, July 07, 2015
In Blinding Eye Disease, Trash-Collecting Cells Go Awry, Accelerate Damage
NIH research points to microglia as potential therapeutic target in retinitis pigmentosa.
Friday, July 03, 2015
Boys More Likely to Have Antipsychotics Prescribed, Regardless of Age
NIH-funded study is the first look at antipsychotic prescriptions patterns in the U.S.
Thursday, July 02, 2015
Potential Therapeutic for Blinding Eye Disease
NIH research points to microglia as potential therapeutic target in retinitis pigmentosa.
Thursday, July 02, 2015
New Medication for Alcohol Use Disorder
NIH begins clinical trial investigating a potential treatment for alcohol use disorder.
Friday, June 26, 2015
Scientific News
Study Finds Brain Chemicals that Keep Wakefulness in Check
Researchers to develop new drugs that promote better sleep, or control hyperactivity in people with mania.
Sorting Through Cellular Statistics
Aaron Dinner, professor in chemistry, and his graduate student Herman Gudjonson are trying to read the manual of life, DNA, as part of the Dinner group’s research into bioinformatics—the application of statistics to biological research.
Playing 'Tag' with Pollution lets Scientists See Who's It
Using a climate model that can tag sources of soot from different global regions and can track where it lands on the Tibetan Plateau, researchers have determined which areas around the plateau contribute the most soot — and where.
Women’s Immune System Genes Operate Differently from Men’s
A new technology reveals that immune system genes switch on and off differently in women and men, and the source of that variation is not primarily in the DNA.
Long Telomeres Associated with Increased Lung Cancer Risk
Genetic predisposition for long telomeres predicts increased lung adenocarcinoma risk.
First Artificial Ribosome Designed
Researchers at the University of Illinois at Chicago and Northwestern University have engineered a tethered ribosome that works nearly as well as the authentic cellular component, or organelle, that produces all the proteins and enzymes within the cell.
High-Resolution 3D Images Reveal the Muscle Mitochondrial Power Grid
NIH mouse study overturns scientific ideas on energy distribution in muscle.
Expanding the Brain
A team of researchers has identified more than 40 new “imprinted” genes, in which either the maternal or paternal copy of a gene is expressed while the other is silenced.
Identifying a Key Growth Factor in Cell Proliferation
Researchers discover that aspartate is a limiter of cell proliferation.
Study Uncovers Target for Preventing Huntington’s Disease
Scientists from Cardiff University believe that a treatment to prevent or delay the symptoms of Huntington’s disease could now be much closer, following a major breakthrough.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!