Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates-Reference-Cited by-同舟云学术

Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates

Published:2019-06 Issue:2 Volume:30 Page:50-56
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Tobias Peters¹²,Philipp Seitz Immanuel¹,Stylianos Michalakis³,Martin Biel³,Barbara Wilhelm²,Felix Reichel¹,Alexander Ochakovski Guy¹,Eberhart Zrenner⁴⁵,Marius Ueffing⁵,Birgit Korbmacher⁶,Sven Korte⁶,Ulrich Bartz-Schmidt Karl¹,Dominik Fischer Manuel¹,Bartz-Schmidt Karl Ulrich,Bolz Sylvia,Fischer Dominik,Kohl Susanne,Kühlewein Laura,Mühlfriedel Regine,Neubauer Jonas,Ochakovski Alex,Paquet-Durand Francois,Seeliger Mathias,Sothilingam Vithiyanjali,Ueffing Marius,Weisschuh Nicole,Wissinger Bernd,Zhour Ahmad,Zobor Ditta,Zrenner Eberhart,Biel Martin,Michalakis Stylianos,Schön Christian,Kahle Nadine,Peters Tobias,Wilhelm Barbara,Tsang Steven,Glöckner Christian Johannes,

Affiliation:

1. University Eye Hospital Tübingen, University of Tübingen, Tübingen, Germany.

2. STZ Eyetrial, University of Tübingen, Tübingen, Germany.

3. Center for Integrated Protein Science Munich, Department of Pharmacy – Center for Drug Research, Ludwig Maximilian University of Munich, Munich, Germany.

4. Werner Reichardt Centre for Integrative Neuroscience, University of Tübingen, Tübingen, Germany.

5. Institute for Ophthalmic Research, Centre for Ophthalmology, University of Tübingen, Tübingen, Germany.

6. Covance Preclinical Services GmbH, Münster, Germany.

Publisher

Mary Ann Liebert Inc

Subject

Genetics(clinical)

Link

https://www.liebertpub.com/doi/pdf/10.1089/humc.2018.188

Reference34 articles.

1. Progressive Loss of Cones in Achromatopsia: An Imaging Study Using Spectral-Domain Optical Coherence Tomography

2. Mutations in the CNGB3 gene encoding the beta-subunit of the cone photoreceptor cGMP-gated channel are responsible for achromatopsia (ACHM3) linked to chromosome 8q21