Immunology of AAV-Mediated Gene Transfer in the Eye.
Immunology of AAV-Mediated Gene Transfer in the Eye.
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DOI：
10.3389/fimmu.2013.00261
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发表时间：
2013
影响因子：
7.3
通讯作者：
Bennett J
中科院分区：
文献类型：
--
作者：
Willett K;Bennett J
眼睛一直处于转基因治疗的最前沿，这主要是由于合适的疾病靶点，解剖学可及性和充分研究的免疫豁免。这些优势促进了研究，最终在几个临床试验和腺相关病毒（AAV）已成为许多眼部治疗的首选载体。临床前和临床研究已经评估了对各种天然存在的和工程化的AAV血清型以及它们递送的转基因的体液和细胞免疫应答，并且这些数据已经与潜在的临床后遗症相关。令人鼓舞的是，在一些研究中，AAV似乎是安全有效的，临床随访超过5年。随着AAV在眼睛中的疾病靶点不断扩大，对免疫安全性的全面和审慎评估至关重要。通过仔细研究，这些技术的发展应该同时告知眼部免疫反应的生物学。
The eye has been at the forefront of translational gene therapy largely owing to suitable disease targets, anatomic accessibility, and well-studied immunologic privilege. These advantages have fostered research culminating in several clinical trials and adeno-associated virus (AAV) has emerged as the vector of choice for many ocular therapies. Pre-clinical and clinical investigations have assessed the humoral and cellular immune responses to a variety of naturally occurring and engineered AAV serotypes as well as their delivered transgenes and these data have been correlated to potential clinical sequelae. Encouragingly, AAV appears safe and effective with clinical follow-up surpassing 5 years in some studies. As disease targets continue to expand for AAV in the eye, thorough and deliberate assessment of immunologic safety is critical. With careful study, the development of these technologies should concurrently inform the biology of the ocular immune response.
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影响因子：
20.3
作者：
Jiang, Haiyan;Couto, Linda B.;Pierce, Glenn F.
通讯作者：
Pierce, Glenn F.
影响因子：
--
作者：
Jacobson, Samuel G.;Cideciyan, Artur V.;Ratnakaram, Ramakrishna;Heon, Elise;Schwartz, Sharon B.;Roman, Alejandro J.;Peden, Marc C.;Aleman, Tomas S.;Boye, Sanford L.;Sumaroka, Alexander;Conlon, Thomas J.;Calcedo, Roberto;Pang, Ji-Jing;Erger, Kirsten E.;Olivares, Melani B.;Mullins, Cristina L.;Swider, Malgorzata;Kaushal, Shalesh;Feuer, William J.;Iannaccone, Alessandro;Fishman, Gerald A.;Stone, Edwin M.;Byrne, Barry J.;Hauswirth, William W.
通讯作者：
Hauswirth, William W.
影响因子：
12.4
作者：
Gargiulo, Annagiusi;Bonetti, Ciro;Surace, Enrico M.
通讯作者：
Surace, Enrico M.
影响因子：
5.1
作者：
Dalkara, D.;Byrne, L. C.;Flannery, J. G.
通讯作者：
Flannery, J. G.
影响因子：
--
作者：
KLEIN, R;KLEIN, BEK;DEMETS, DL
通讯作者：
DEMETS, DL