netbook
desktop
mobile
tablet-landscape
tablet
phone-landscape
phone
Research to Prevent Blindness

Renewed Hope for Gene Therapy to Restore Vision

A few years ago, in a major milestone, RPB-supported researchers used gene therapy to restore partial sight in patients with Lebers Congenital Amaurosis, a form of retinitis pigmentosa. For a while, their sight improved. But, earlier this year, reports indicated that the treatment's effects were wearing off, and that the patients' sight was again diminishing as the degeneration of their photoreceptor cells progressed.

One proposed explanation for the ongoing degeneration was that the disease had already reached a point of no return when the initial gene therapy had been applied; the photoreceptors were already beyond permanent rescue.

Now, new findings from the lab of RPB researcher Stephen H. Tsang, MD, PhD, Columbia University College of Physicians & Surgeons, are rekindling hope for the use of gene therapy to restore vision in patients with retinal degenerative conditions.

"Our report is the first to demonstrate a stable halting of a retinal degeneration," says Tsang. "It also demonstrates a relatively broad therapeutic time window for a preclinical retinitis pigmentosa (RP) model."

The Tsang team found that, if the gene therapy was optimally delivered, it could be effective at any time during the course of the disease.

"Our study points to non-optimal delivery of the gene therapeutic as a cause of continued degeneration, and suggests that the way forward for developing RP therapeutics is to focus on optimizing delivery, which we have demonstrated in a model. Our work also speaks to the need for earlier precision medicine diagnosis, so these gene therapeutics can freeze the degeneration cascade earlier in the disease course." (Read a Profile on RPB Physician-Scientist Tsang, his work on precision diagnosis, and the influence that RPB Grants have had on his career.)

According to Tsang, the results of this project will allow gene therapists to better counsel their patients on prognosis, alert them to critical periods for treatment and assess the efficacy of future therapeutic approaches.

Most important to patients, these findings suggest that RP can be treated despite being diagnosed after the disease has compromised many cells.

Related News: Drug Delivery, Feature Story, Gene Therapy, Retinitis Pigmentosa, Top Story

Dr. Semenza and colleague work in his lab

Research to Prevent Blindness Grantee Dr. Gregg Semenza Wins Nobel Prize in Medicine

Dr. Semenza is being recognized for his discoveries related to how cells respond to low oxygen levels, which have implications for the treatment of cancer, eye diseases and more.

Read More

 
RPB logo

RPB and Partners Award $1.2 Million In Grants for Novel AMD Research

Four new grantees received the Catalyst Awards for Innovative Research Approaches for AMD.

Read More

 
CDA 30th Anniversary book

The Career Development Award Turns 30: Celebrating a Generation of Support for Early-Career Scientists

CDA founded 30 years ago as one of the few private sources of funding aimed specifically at early-career vision researchers.

Read More

 
Retinal image of person with Alzheimer's disease

Could An Eye Exam Reveal Alzheimer’s Disease?

Study from RPB-supported researchers at Duke University suggests loss of blood vessels in retina reflect changes in brain health.

Read More

 
Gregory Schwartz in the lab

New Findings on Blood Vessel Regulation

A study from an RPB-supported researcher at Northwestern University School of Medicine could speed up the diagnosis of retinal diseases.

Read More

 
NEI Audacious Goals image and text

RPB-SUPPORTED RESEARCHERS INTEGRAL TO NEI’S AUDACIOUS GOALS INITIATIVE

Vision scientists who have received RPB funding will help lead all five research teams, which will seek to accelerate development of regenerative treatments for blindness.

Read More

 

Subscribe

Get our email updates filled with the latest news from our researchers about preventing vision loss, treating eye disease and even restoring sight. Unsubscribe at any time. Under our privacy policy, we'll never share your contact information with a third party.