Institute for Ophthalmic Research
Centre for Ophthalmology,
University of Tübingen
Elfriede-Aulhorn-Strasse 7
D-72076 Tübingen,
Germany
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Selecting the right patients for gene-based therapies: Development and implementation of a pathogenicity scoring system combined with functional in vitro validation of gene variants and genotypes in patients with inherited retinal dystrophies as a criterion for the eligibility for therapeutic studies
Selecting the right patients for gene-based therapies: Development and implementation of a pathogenicity scoring system combined with functional in vitro and in vivo validation of gene variants and genotypes in patients with inherited retinal dystrophies as a criterium for the eligibility for therapeutic studies
Stylianos Michalakis
Stefan Liebau
Kevin Achberger
Kerstin Nagel-Wolfrum