New eye gene therapy close to clinical...

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New eye gene therapy close to clinical testing

There are many different retinal diseases simply because many different steps of the visual process can be affected. However, they all have one thing in common: correction of the relevant defective gene currently provides the only possibility of permanent cure. Prof. Dr. Mathias Seeliger and his group of researchers at the Institute of Ophthalmic Research at the University Hospital in Tübingen is specifically focused on the development of gene therapies for the treatment of neurodegenerative eye diseases. The techniques, developed in cooperation with colleagues from the University of Munich, have been lastingly successful in mice. A gene therapy for the treatment of this kind of retinal disease is now close to clinical testing.

Prof. Dr. Mathias Seeliger has been specifically working on congenital retinal diseases for more than 15 years. Seeliger is an engineer, medical doctor and head of the ocular neurodegeneration research group at the Institute of Ophthalmic Research at the University of Tübingen. Amongst other things, Seeliger’s work involves congenital retinal diseases. Patients with congenital retinal diseases exhibit modifications in the genes that are responsible for the proper function of the retinal cells.

Most of these modifications are point mutations which, depending on where they occur in the DNA strand, may lead to the complete termination of protein synthesis, a lower than normal expression of the relevant protein in the cell or the production of a faulty 'disturbing' protein. All this affects retinal vision and results in a number of serious eye diseases.

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