Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes

Author:

Grotz Sophia12,Schäfer Jessica3,Wunderlich Kirsten A3,Ellederova Zdenka4,Auch Hannah12,Bähr Andrea25,Runa‐Vochozkova Petra5ORCID,Fadl Janet13,Arnold Vanessa3,Ardan Taras4,Veith Miroslav6ORCID,Santamaria Gianluca5,Dhom Georg12,Hitzl Wolfgang7ORCID,Kessler Barbara12ORCID,Eckardt Christian25,Klein Joshua3,Brymova Anna4,Linnert Joshua3,Kurome Mayuko12,Zakharchenko Valeri12,Fischer Andrea8,Blutke Andreas9,Döring Anna8,Suchankova Stepanka10,Popelar Jiri10,Rodríguez‐Bocanegra Eduardo1112,Dlugaiczyk Julia13,Straka Hans14,May‐Simera Helen15ORCID,Wang Weiwei16ORCID,Laugwitz Karl‐Ludwig5,Vandenberghe Luk H16,Wolf Eckhard12ORCID,Nagel‐Wolfrum Kerstin317,Peters Tobias1112ORCID,Motlik Jan4,Fischer M Dominik1819,Wolfrum Uwe3ORCID,Klymiuk Nikolai25ORCID

Affiliation:

1. Chair of Molecular Animal Breeding and Biotechnology LMU Munich Munich Germany

2. Center for Innovative Medical Models LMU Munich Munich Germany

3. Institute of Molecular Physiology Molecular Cell Biology Johannes Gutenberg University (JGU) Mainz Germany

4. Institute of Animal Physiology and Genetics Czech Academy of Science Libechov Czech Republic

5. Large Animal Models in Cardiovascular Research Internal Medical Department I TU Munich Munich Germany

6. Ophthalmology Clinic University Hospital Kralovske Vinohrady Praha Czech Republic

7. Biostatistics and Data Science Paracelsus Medical University Salzburg Austria

8. Veterinary Faculty Small Animal Clinics LMU Munich Munich Germany

9. Institute of Experimental Genetics Helmholtz Center Munich Neuherberg Germany

10. Institute of Experimental Medicine Czech Academy of Science Prague Czech Republic

11. Centre for Ophthalmology University Eye Hospital University Hospital Tübingen Tübingen Germany

12. Institute for Ophthalmic Research Centre for Ophthalmology University Hospital Tübingen Tübingen Germany

13. Department of Otorhinolaryngology, Head and Neck Surgery University Hospital Zurich (USZ) University of Zurich Zurich Switzerland

14. Faculty of Biology LMU Munich Planegg Germany

15. Institute of Molecular Physiology Cilia Biology JGU Mainz Mainz Germany

16. Grousbeck Gene Therapy Center Mass Eye and Ear and Harvard Medical School Boston MA USA

17. Institute of Developmental Biology and Neurobiology Johannes Gutenberg University (JGU) Mainz Germany

18. Oxford Eye Hospital Oxford University NHS Foundation Trust Oxford UK

19. Nuffield Laboratory of Ophthalmology NDCN University of Oxford Oxford UK

Funder

Deutsche Forschungsgemeinschaft

Foundation Fighting Blindness

Publisher

EMBO

Subject

Molecular Medicine

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