Gene Therapy: First success and many...

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Gene Therapy: First success and many unanswered questions

Prof. Marius Ueffing, Director of the Institute for Ophthalmic Research in Tuebingen explains translational approaches for restoring sight

In the Weekly German Medical Review (Deutsche Medizinische Wochenschrift) Dr. Ueffing exemplifies the present situation of gene therapeutic approaches in ophthalmology.

The goal is to develop gene and cell therapy for sight loss by translating basic research into new treatments. With this new kind of therapy researchers hope to reverse some vision loss in people born with a degenerative eye disease for which there is no existing treatment. The treatment adds a working copy of the culpable gene to the retinal cells of patients born with a defective copy.