Safety and vision outcomes of subretinal gene supplementation therapy in <i>PDE6A</i> -associated retinitis pigmentosa: a non-randomised controlled trial
Subretinal gene therapy with AAV8.hPDE6A did not improve visual function over 1 year and posed risks, including central retinal thinning and visual acuity decline. This is in contrast to the safety and efficacy profile established in preclinical models.
Authors
- M. Ziégler (ORCID: https://orcid.org/0000-0002-6229-5698)
- Stylianos Michalakis (ORCID: https://orcid.org/0000-0001-5092-9238)
- Susanne Kohl (ORCID: https://orcid.org/0000-0002-6438-6331)
- Nicole Weisschuh (ORCID: https://orcid.org/0000-0002-1047-6227)
- Marius Ueffing (ORCID: https://orcid.org/0000-0003-2209-2113)
- Stephen H. Tsang (ORCID: https://orcid.org/0000-0001-9082-2427)
- Immanuel P. Seitz (ORCID: https://orcid.org/0000-0002-3086-6425)
- Regine Muehlfriedel (ORCID: https://orcid.org/0000-0001-7955-1654)
- Eberhart Zrenner (ORCID: https://orcid.org/0000-0003-2846-9663)
- Barbara Wilhelm (ORCID: https://orcid.org/0000-0002-4289-1353)
- Laura Kuehlewein (ORCID: https://orcid.org/0000-0003-1906-8681)
- Peter Martus (ORCID: https://orcid.org/0000-0002-5386-5732)
- Felix F. Reichel (ORCID: https://orcid.org/0000-0001-9325-5616)
- Bernd Wissinger
- François Paquet‐Durand (ORCID: https://orcid.org/0000-0001-7355-5742)
- Katarína Štingl (ORCID: https://orcid.org/0000-0002-8132-911X)
- Mathias W. Seeliger (ORCID: https://orcid.org/0000-0002-7879-6788)
- Martin Biel (ORCID: https://orcid.org/0000-0002-9974-3052)
- Tobias Peters (ORCID: https://orcid.org/0000-0002-9187-4904)
- Karl Ulrich Bartz-Schmidt
- M. Dominik Fischer (ORCID: https://orcid.org/0000-0002-0690-6538)
Institutions
- LMU Klinikum (DE)
- University of Oxford (GB)
- University Children's Hospital Tübingen (DE)
- STZ eyetrial (DE)
- Columbia University (US)
- University of Tübingen (DE)
- Ludwig-Maximilians-Universität München (DE)
Publication Details
- Journal
- British Journal of Ophthalmology
- Published
- 2025-08-18
- DOI
- https://doi.org/10.1136/bjo-2024-326480
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Tistou and Charlotte Kerstan Stiftung