"Endogena Therapeutics has dosed the first patient in its Phase 1/2 clinical trial for E-2353, its small molecule designed to activate dormant retinal stem cells in the eyes of patients with retinitis pigmentosa (RP) to produce photoreceptors, the retinal cells that make vision possible, but degenerate in RP. The endogenous retinal stem cells migrate from the ciliary epithelium, located just behind the iris, to the retina, where they develop into photoreceptors. The treatment is designed to work regardless of the gene causing the RP."
An innovative, risk-free technique that could one day be used to correct genetic faults has been reported by a group of scientists from the University of California, San Diego.
The team is comprised of professor Ethan Bier, postdoctoral researcher Sitara Roy, and expert Annabel Guichard, according to ANI's report.
Their strategy makes use of the body's internal DNA repair systems, which ultimately provide a pathway for advanced gene therapy techniques that could treat a variety of genetic problems.
The new study represents a step forward in an effort to bring photoreceptor transplantations to patients with blinding diseases. “To our knowledge, this is the first time someone has achieved such a massive integration of transplanted photoreceptors into the retina,” says Prof. Marius Ader, research group leader at the Center for Regenerative Therapies Dresden (CRTD) at TU Dresden who led the study.
A new study reveals how photoreceptors grown from stem cells might extend biological wires, known as axons, to contact existing neurons.
The finding has implications for future treatment of retinal diseases that cause blindness, including age-related macular degeneration and rare diseases such as retinitis pigmentosa, Usher syndrome, Stargardt disease and Best disease.
"The results of this study confirm the ability of lutein to postpone photoreceptor degeneration by reducing reactive gliosis of Müller cells in the retina and exerting anti-inflammatory effects."
"Noting recent research into the role of hormones, particularly progesterone, in preventing cell death due to oxidative stress, the CEU UCH Drug Delivery Systems(DDS)research group has successfully developed and tested a range of methods of delivery of this hormone into the eye to slow the degenerative process characteristic of retinitis pigmentosa."
Optogenetics holds promise for restoring lost vision. In Usher 1F, the cells that die because of the genetic mutation are photoreceptor cells. The layer of cells in the retina below the photo receptors are the retinal ganglion cells. These are not affected Usher 1F. Optogenetics delivers a compound to the ganglion cells to convert them to photoreceptor cells. Some vision restoration is achieved.
What is notable about this article is that one of the authors is Jean Bennett, the pioneer of gene therapy for inherited retinal diseases - She is the one who developed the gene therapy, Luxturna, that is now approved for one type of LCA, a severe form of RP.
"Researchers have identified distinct differences among the cells comprising a tissue in the retina that is vital to human visual perception. The scientists discovered five subpopulations of retinal pigment epithelium (RPE) -- a layer of tissue that nourishes and supports the retina's light-sensing photoreceptors. Using artificial intelligence, the researchers analyzed images of RPE at single-cell resolution to create a reference map that locates each subpopulation within the eye."
"The development of alternative, non-viral delivery platforms like nanoparticles is of great interest to extend the application of gene therapy for RP. Amino-functionalized mesoporous silica-based nanoparticles (N-MSiNPs) were synthesized, physico-chemically characterized, and evaluated as gene delivery systems for human cells in vitro and for retinal cells in vivo. .... No adverse effects were detected for the integrity of the retinal tissue or the visual function of treated eyes. N-MSiNPs were able to deliver a therapeutic transgene candidate for RP, PRPF31, both in vitro and in vivo. N-MSiNPs are safe for retinal delivery and thus a potential alternative to viral vectors."
"Vinberg points out this approach can reduce research costs compared to non-human primate research and dependence on animal models that produce results that do not always apply to humans. While mice are commonly used in vision research, they do not have a macula. Researchers can also test potential new therapies on functioning human eye cells, speeding drug development."