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Are There Limits to What Gene Therapy Can Achieve in Treating Retinal Blindness?

New Rochelle, NY, March 8, 2013— Follow-up results from one of several groundbreaking gene therapy studies reported in 2008 in patients with an inherited form of retinal blindness confirmed the earlier positive results of substantial improvement in vision that has now persisted for more than 4 years. However, the new study* also showed that while gene therapy was able to improve patients' vision, it did not slow or stop the underlying disease process.

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RetroSense Therapeutics Announces Notice of Allowance for New U.S. Patent Application Broadly Covering Optogenetic Approaches to Vision Restoration

RetroSense Therapeutics, a biotechnology company dedicated to developing gene therapy approaches to vision restoration, announced today that the U.S. Patent and Trademark Office has issued a Notice of Allowance for U.S. patent application (No. 12/299,574), which broadly covers methods of restoring visual responses with a variety of optogenetic compounds.

Researchers show that lipid nanoparticles are ideal for delivering genes and drugs

UPV/EHU-University of the Basque Country researchers are using nanotechnology for therapeutic purposes.

This shows the expression of the retinoschisis protein (green) after the transfection of a cell line of retina pigment epithelium (ARPE-19) with a lipid nanoparticle-based formulation with the RS1 gene....

Growth factor TGF-B helps maintain health of retinal blood vessels

Scientists at Schepens Eye Research Institute have found that the growth factor known as TGF-beta is essential to the health of blood vessels in the retina and that blocking it can cause retinal dysfunction. These findings, published in the April 2 issue of PLoS ONE, may have an important impact on the prevention and treatment of diseases such as diabetic retinopathy and macular degeneration.

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