NIH-funded therapy will now be tested in humans.
"Nanoscope’s findings, reported today in Nature Gene Therapy, show that totally blind mice—meaning they have no light perception—regain significant retinal function and vision after treatment. Studies described in the report showed that treated mice were significantly faster in standardized visual tests, such as navigating mazes and detecting changes in motion."
Proof of concept for LCA, another inherited retinal disease, using base editing. This tool was developed by David Liu, PhD, Harvard, who is working with our Harvard Medical School researcher, David Corey, PhD, to develop the same for Usher 1F.
"The first reported clinical results of a Phase I study following implantation of the NR600 Implant (Nano Retina Ltd.) indicated that this innovative three-dimensional neural interface retinal prosthesis is safe in patients with end-stage retinitis pigmentosa (RP), defined as no-to-minimal light perception.
"The secondary endpoint, i.e., visual capabilities after the device was implanted, was also met, according to Dr Peter Stalmans, , a vitreoretinal surgeon from the Ophthalmology Department, University Hospitals Leuven, Belgium."
“'It is impressive to see how patients that have lived for more than 5 years in complete darkness are able again to self-walk and can find and recognise objects around them,' Dr Stalmans stated."
"Simvastatin is a 3‐hydroxy‐3‐methylglutaryl CoA reductase inhibitor with multiple targets and effects. It protects neurons in the brain, but its protective effects on photoreceptors are unclear. In this study, we evaluated the neuroprotective effect of simvastatin on photoreceptors exposed to stress induced by all‐trans‐retinal (atRAL)....Our findings suggest that simvastatin has a novel role in protecting photoreceptors from atRAL‐induced stress. Simvastatin treatment resulted in up‐regulation of IRBP and its upstream transcription factor CRX in Y79 cells, ex vivo human retinal explants, and murine retinas in vivo. Further studies of simvastatin to treat photoreceptor degeneration are warranted."
"Ocular inflammation is a common pathological condition of a series of retinal degenerative diseases. Tetramethylpyrazine (TMP), a Chinese herbal extraction, is widely used in the treatment of several ocular diseases in Eastern countries. However, the exact mechanisms correlating the vision protective effects of TMP have not been elucidated. Thus, this study aimed to investigate TMP’s molecular targets in anti-inflammatory activity. ... Therefore, this study elucidated a novel mechanism that TMP inhibits retinal inflammation by inhibiting microglial activation via a TLR4/NF-κB signalling pathway."
In the recent years, natural compounds have shown great pharmacological potentials. Pentacyclic triterpenoids (PTs) are a group of compounds widely existing in nature, they have shown diverse pharmacological effects in vitro and in vivo. In this thesis, we evaluated the antioxidative effect of betulin, betulinic acid and their 18 derivatives in human retinal pigment epithelium (RPE) and human Müller cells.
"Excitotoxicity seems to play a critical role in ocular neurodegeneration. Excess-glutamate-mediated retinal ganglion cells death is the principal cause of cell loss. Uncontrolled glutamate in the synapsis has significant implications in the pathogenesis of neurodegenerative disorders. The exploitation of various approaches of controlled release systems enhances the pharmacokinetic and pharmacodynamic activity of drugs. In particular, microparticles are secure, can maintain therapeutic drug concentrations in the eye for prolonged periods, and make intimate contact by improving drug bioavailability. According to the promising results reported, possible new investigations will focus intense attention on microparticulate formulations and can be expected to open the field to new alternatives for doctors, as currently required by patients."
"Laser based 'naked DNA gene therapy' in a spatially targeted manner will pave the way for treatment of inherited retinal diseases."
"Eyevensys is developing EYS611, a treatment for Retinitis Pigmentosa and potentially other retinal degenerative conditions including the later stages of Dry AMD and glaucoma. The treatment encodes for a potent iron chelator with antioxidant and endogenous neuroprotective properties. In animal models, the treatment has been shown to be safe and effective at preserving and slowing the degeneration of retinal structure and function."
Stem cell therapy showing promise, regardless of RP genotype.
"In study, treatment demonstrates promising biologic activity, safety profile."