The UK Medicines and Healthcare Regulatory Agency (MHRA) has accepted GenSight’s application to run a first clinical trial in the UK testing a new type of therapy with the potential to treat all forms of retinitis pigmentosa. ...The Phase I/II trial, called PIONEER, will study the safety and tolerability of GenSight’s therapy, called GS030, in patients with end-stage retinitis pigmentosa with vision not better than “counting fingers.” The company expects to treat the first patient in the first quarter of 2018 and will measure the outcome after a year.
"MANF (mesencephalic-astrocyte-derived neurotrophic factor) is believed to have broad potential because it is a naturally-occurring protein produced by the body to reduce/prevent apoptosis (cell death) in response to injury or disease, via the unfolded protein response...MANF's lead indication is retinitis pigmentosa."
A powerful purple protein that could help cure certain types of blindness has made its way from a tiny Farmington laboratory all the way up to the International Space Station.
FINDINGS In this case-control study of children with retinitis pigmentosa, including 55 taking an age-adjusted dose of vitamin A (5000-15 000 IU/d) and 25 not taking vitamin A, vitamin A supplementation was associated with almost 50% slowing of the mean exponential rate of decline of full-field cone electroretinogram amplitude.
MEANING These findings, while not definitive in the absence of randomized controls, support the hypothesis that vitamin A palmitate can slow loss of cone function in children with common forms of retinitis pigmentosa.
"For the purpose of their work, they invented a kind of "improved CRISPR," which they say cuts more precisely."
"A simple retinal prosthesis is under development by Tel Aviv University in Israel and Linköping University in Sweden. Fabricated using cheap and widely available organic pigments used in printing inks and cosmetics, it consists of tiny pixels like a digital camera sensor on a nanometric scale. The researchers hope that it can restore sight to blind people."
"Researchers revealed that culturing human induced pluripotent stem cells with different isoforms of the extracellular component laminin led to the creation of cells specific to different parts of the eye, including retinal, corneal, and neural crest cells. They showed that the different laminin variants affected the cells' motility, density, and interactions, resulting in their differentiation into specific ocular cell lineages. Cells cultured in this way could be used to treat various ocular diseases."
"Scientists at the Francis Crick Institute have discovered a set of simple rules that determine the precision of CRISPR/Cas9 genome editing in human cells. These rules, published in Molecular Cell, could help to improve the efficiency and safety of genome editing in both the lab and the clinic."
"The US Food & Drug Administration (FDA) has given the go-ahead for clinical testing of what could be the first gene editing treatment to be used in inherited eye disease. The new treatment, known as EDIT-101, has been developed by US-based pharmaceutical company Editas Medicine and aims to use the relatively new gene editing technique known as CRISPR to treat Leber Congenital Amaurosis type 10 (LCA10)."
"While there is still a lot of research to be done—at least 10 years' worth, first on animals and then on humans—this technology could make all the difference in an aging population suffering from eye deterioration for which there are still no effective long-term treatments. It also has the advantage of avoiding the use of viruses commonly employed in gene therapy. These researchers are looking at applications of this technology in all eye diseases, but more particularly in glaucoma, retinitis and macular degeneration."