Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia-Reference-Cited by-同舟云学术

Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia

Published:2020-06-01 Issue:6 Volume:138 Page:643
ISSN:2168-6165
Container-title:JAMA Ophthalmology
language:en
Short-container-title:JAMA Ophthalmol

Author:

Fischer M. Dominik¹²³,Michalakis Stylianos⁴⁵,Wilhelm Barbara³,Zobor Ditta²,Muehlfriedel Regine²,Kohl Susanne²,Weisschuh Nicole²,Ochakovski G. Alex¹,Klein Reinhild⁶,Schoen Christian⁴,Sothilingam Vithiyanjali²,Garcia-Garrido Marina²,Kuehlewein Laura¹,Kahle Nadine¹³,Werner Annette²,Dauletbekov Daniyar¹²,Paquet-Durand François²,Tsang Stephen⁷,Martus Peter⁸,Peters Tobias³,Seeliger Mathias²,Bartz-Schmidt Karl Ulrich¹,Ueffing Marius²,Zrenner Eberhart¹²,Biel Martin⁴,Wissinger Bernd²

Affiliation:

1. University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany

2. Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany

3. Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany

4. Center for Integrated Protein Science Munich, Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-University of Munich, Munich, Germany

5. Department of Ophthalmology, Ludwig-Maximilians-Universität München, Munich, Germany

6. Department of Internal Medicine II, University Hospital Tübingen, Germany

7. Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, New York

8. Institute for Clinical Epidemiology and Applied Biostatistics, University Hospital Tübingen, Tübingen, Germany

Publisher

American Medical Association (AMA)

Subject

Ophthalmology

Link

https://jamanetwork.com/journals/jamaophthalmology/articlepdf/2765029/jamaophthalmology_fischer_2020_oi_200028.pdf

Reference33 articles.

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2. Retinal structure and function in achromatopsia: implications for gene therapy.;Sundaram;Ophthalmology,2014

3. Progressive loss of cones in achromatopsia: an imaging study using spectral-domain optical coherence tomography.;Thiadens;Invest Ophthalmol Vis Sci,2010

4. The clinical phenotype of CNGA3-related achromatopsia: pretreatment characterization in preparation of a gene replacement therapy trial.;Zobor;Invest Ophthalmol Vis Sci,2017

5. Causes and consequences of inherited cone disorders.;Roosing;Prog Retin Eye Res,2014

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