Retinitis pigmentosa tagged posts

Gene therapy may be the best hope for curing retinitis pigmentosa (RP), an inherited condition that usually leads to severe vision loss and blinds 1.5 million people worldwide.

But there’s a huge obstacle: RP can be caused by mutations in over 80 different genes. To treat most RP patients with gene therapy, researchers would have to create a therapy for each gene—a nearly impractical task using current gene therapy strategies.

A more universal treatment may be forthcoming...

The left side of the figure illustrates the retina transplantation procedure. The right side of the figure represents examples of orientation tuning curves recorded from three rats: a normal rat, a rat with a transplant and a rat with a degenerated retina (blind). Displayed below the tuning curves are the responses to sinusoidal grating stimuli. The response patterns indicate that neurons in the brains of transplant recipients are very similar to those of the rats with normal vision.

Researchers from the University of California, Irvine School of Medicine, have discovered that neurons located in the vision centers of the brains of blind rats functioned normally following fetal retina cell transplants, indicating the successful restoration of vision...

An artist’s rendering incorporates the images of the Müller glia-derived rod photoreceptors. These photoreceptors were structurally no different from real photoreceptors and they became integrated within the circuitry of the visual pathway, from the retina to the brain. Credit: Bo Chen, Ph.D.

New technique generates rod photoreceptors that integrate into the retina and brain. Researchers funded by the National Eye Institute (NEI) have reversed congenital blindness in mice by changing supportive cells in the retina called Müller glia into rod photoreceptors. The findings advance efforts toward regenerative therapies for blinding diseases such as age-related macular degeneration and retinitis pigmentosa.

“This is the first report of scientists reprogramming Müller glia to become functional...

Correction of RPGR mutation in patient iPSC line.

CUMC and Uni of Iowa scientists have used CRISPR, to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide. It marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells that were derived from a patient’s tissue.

“Our vision is to develop a personalized approach to treating eye disease,” says A/Prof Stephen Tsang, MD, PhD. “We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease. Here we have demonstrated that the initial steps are feasible...