AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye

被引：110

作者：

Reichel, Felix F. ^{[1
,2
]}

Dauletbekov, Daniyar L. ^{[1
,2
]}

Klein, Reinhild ^{[3
]}

Peters, Tobias ^{[2
,4
]}

Ochakoyski, G. Alex ^{[1
,2
]}

Seitz, Immanuel P. ^{[1
,2
]}

Wilhelm, Barbara ^{[2
,4
]}

Ueffing, Marius ^{[2
]}

Biel, Martin ^{[5
]}

Wissinger, Bernd ^{[2
]}

Michalakis, Stylianos ^{[5
]}

Bartz-Schmidt, Karl Ulrich ^{[1
]}

Fischer, M. Dominik ^{[1
,2
,4
,6
]}

机构：

[1] Univ Hosp Tubingen, Ctr Ophthalmol, Univ Eye Hosp, Tubingen, Germany

[2] Univ Hosp Tubingen, Ctr Ophthalmol, Inst Ophthalm Res, Tubingen, Germany

[3] Univ Hosp Tubingen, Dept Internal Med 2, Immunopathol Lab, Tubingen, Germany

[4] Univ Hosp Tubingen, STZ Eyetrial Ctr Ophthalmol, Tubingen, Germany

[5] Ludwig Maximilians Univ Munchen, Ctr Drug Res, Dept Pharm, CIPSM, Munich, Germany

[6] Univ Oxford, Nuffield Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England

来源：

MOLECULAR THERAPY | 2017年 / 25卷 / 12期

关键词：

LEBERS CONGENITAL AMAUROSIS; RETINAL GENE-THERAPY; SUBRETINAL INJECTION; VISUAL FUNCTION; LONG-TERM; IN-VITRO; ACHROMATOPSIA; PROFILES; VECTOR; SYSTEM;

D O I：

10.1016/j.ymthe.2017.08.018

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Ocular gene therapy has evolved rapidly into the clinical realm due to promising pre-clinical proof-of-concept studies, recognition of the high unmet medical need of blinding disorders, and the excellent safety profile of the most commonly used vector system, the adeno-associated virus (AAV). With several trials exposing subjects to AAV, investigators independently report about cases with clinically evident inflammation in treated eyes despite the concept of ocular immune privilege. Here, we provide a detailed analysis of innate and adaptive immune response to clinical-grade AAV8 in non-human primates and compare this to preliminary clinical data from a retinal gene therapy trial for CNGA3-based achromatopsia.

引用

页码：2648 / 2660

页数：13

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