Tobias, Peters; Philipp, Seitz Immanuel; Stylianos, Michalakis; Martin, Biel; Barbara, Wilhelm; Felix, Reichel; Alexander, Ochakovski Guy; Eberhart, Zrenner; Marius, Ueffing; Birgit, Korbmacher; Sven, Korte; Ulrich, Bartz-Schmidt Karl; Dominik, Fischer Manuel; Bartz-Schmidt, Karl Ulrich; Bolz, Sylvia; Fischer, Dominik; Kohl, Susanne; Kuehlewein, Laura; Muehlfriedel, 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; Gloeckner, Christian Johannes (2019): Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates. In: Human Gene Therapy Clinical Development, Vol. 30, No. 2: pp. 50-56 |
Abstract
The purpose of this study was to examine the toxicity and side effects of a recombinant adeno-associated virus 8 (AAV8) vector, aimed to treat cyclic nucleotide gated channel alpha 3 (CNGA3)-linked achromatopsia, after a single subretinal administration in cynomolgus macaques. Animals were followed in two studies: a 13-week study with 22 animals and a 28-day study with 12 animals. Both groups were divided into subgroups receiving either vehicle only, a low (1 x 10(11) vector genomes (vg)), or a high dose (1 x 10(12) vg) of rAAV.hCNGA3. In the 13-week study, an extra group received single high-dose intravitreal injections. Here we present the group results of the histological examinations carried out after necropsy from the 28-day study, the retinal functional (electroretinography) in the 13-week study, and clinical observations from both studies. Treatment-related adverse effects were not found, and parameter changes were mostly related to the surgical procedure. The treatment of achromatopsia with rAAV.hCNGA3 is therefore deemed safe to apply to humans.
Item Type: | Journal article |
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Faculties: | Chemistry and Pharmacy > Department of Pharmacy |
Subjects: | 500 Science > 540 Chemistry |
ISSN: | 2324-8637 |
Language: | English |
ID Code: | 83621 |
Deposited On: | 15. Dec 2021 15:08 |
Last Modified: | 15. Dec 2021 15:08 |
- BASE
- Tobias, Peters
- Philipp, Seitz Immanuel
- Stylianos, Michalakis
- Martin, Biel
- Barbara, Wilhelm
- Felix, Reichel
- Alexander, Ochakovski Guy
- Eberhart, Zrenner
- Marius, Ueffing
- Birgit, Korbmacher
- Sven, Korte
- Ulrich, Bartz-Schmidt Karl
- Dominik, Fischer Manuel
- Bartz-Schmidt, Karl Ulrich
- Bolz, Sylvia
- Fischer, Dominik
- Kohl, Susanne
- Kuehlewein, Laura
- Muehlfriedel, 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
- Gloeckner, Christian Johannes
- Google Scholar
- Tobias, Peters
- Philipp, Seitz Immanuel
- Stylianos, Michalakis
- Martin, Biel
- Barbara, Wilhelm
- Felix, Reichel
- Alexander, Ochakovski Guy
- Eberhart, Zrenner
- Marius, Ueffing
- Birgit, Korbmacher
- Sven, Korte
- Ulrich, Bartz-Schmidt Karl
- Dominik, Fischer Manuel
- Bartz-Schmidt, Karl Ulrich
- Bolz, Sylvia
- Fischer, Dominik
- Kohl, Susanne
- Kuehlewein, Laura
- Muehlfriedel, 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
- Gloeckner, Christian Johannes