Surgical Aspects in Gene Therapy for Inherited Retinal Diseases

M. Dominik Fischer; Karl Ulrich Bartz-Schmidt; Spyridon Dimopoulos; Philipp Herrmann; Maximilian Gerhardt; Frank G. Holz; Siegfried Priglinger

doi:10.1055/a-1315-1331

Klinische Monatsblätter für Augenheilkunde, Table of Contents

Klin Monbl Augenheilkd 2021; 238(03): 267-271
DOI: 10.1055/a-1315-1331

Übersicht

Surgical Aspects in Gene Therapy for Inherited Retinal Diseases

Article in several languages: English | deutsch

Authors

M. Dominik Fischer‡

¹Department für Augenheilkunde, Universitäts-Augenklinik, Universitätsklinikum Tübingen, Deutschland

²Nuffield Laboratory of Ophthalmology, University of Oxford, United Kingdom of Great Britain and Northern Ireland
Karl Ulrich Bartz-Schmidt‡

¹Department für Augenheilkunde, Universitäts-Augenklinik, Universitätsklinikum Tübingen, Deutschland
Spyridon Dimopoulos

¹Department für Augenheilkunde, Universitäts-Augenklinik, Universitätsklinikum Tübingen, Deutschland
Philipp Herrmann

³Augenklinik, Universitätsklinikum Bonn, Deutschland
Maximilian Gerhardt

⁴Augenklinik der LMU, Klinikum der Universität München, Deutschland
Frank G. Holz

³Augenklinik, Universitätsklinikum Bonn, Deutschland
Siegfried Priglinger

⁴Augenklinik der LMU, Klinikum der Universität München, Deutschland

Abstract

Inherited retinal dystrophies (IRD) have been studied since their recognition by Franz Donders and Albrecht von Graefe. It nevertheless took 100 years for a causal therapy to take shape in the form of gene therapy: The approval of Voretigen Neparvovec (VN) for the treatment of hereditary retinal dystrophies due to RPE65 mutations was thus a significant milestone – for the era of personalised medicine in general and ophthalmology in particular. The clinical management around gene therapy applications is complex and requires the cooperation of various experts as a multidisciplinary team. This article describes the requirements, challenges, approaches, and open questions regarding the surgical aspects of gene therapy for retinal dystrophies. The first part outlines the standard surgical treatment. Based on this standard, alternative approaches are indicated for each individual step and their value discussed. Knowledge gaps are defined and in the outlook we speculate on future developments.

Key words

retina - dystrophy - gene therapy

Full Text

References

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