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Ocular therapies for neuronal ceroid lipofuscinoses: more than meets the eye.

作者信息

Murray Samantha J, Mitchell Nadia L

机构信息

Faculty of Agricultural and Life Sciences, Lincoln University, Lincoln, New Zealand.

出版信息

Neural Regen Res. 2022 Aug;17(8):1755-1756. doi: 10.4103/1673-5374.332148.

DOI:10.4103/1673-5374.332148
PMID:35017433
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8820703/
Abstract
摘要

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Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease.玻璃体内基因治疗可预防 CLN5 神经鞘脂贮积症羊的视网膜功能障碍和变性。
Exp Eye Res. 2021 Jun;207:108600. doi: 10.1016/j.exer.2021.108600. Epub 2021 Apr 28.
2
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.向颅内递送AAV9基因疗法可部分预防CLN6型贝敦氏病小鼠的视网膜变性和视觉缺陷。
Mol Ther Methods Clin Dev. 2021 Jan 5;20:497-507. doi: 10.1016/j.omtm.2020.12.014. eCollection 2021 Mar 12.
3
Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis.玻璃体内酶替代治疗可保存犬 CLN2 神经元蜡样脂褐质沉积症的视网膜结构和功能。
Exp Eye Res. 2020 Aug;197:108130. doi: 10.1016/j.exer.2020.108130. Epub 2020 Jul 1.
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Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.基因治疗靶向内视网膜可挽救 CLN3 神经鞘脂贮积病小鼠模型的视网膜表型。
Hum Gene Ther. 2020 Jul;31(13-14):709-718. doi: 10.1089/hum.2020.038.
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A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.经枕大池蛛网膜下腔腔隙安全可靠地递送达神经中枢的 AAV 载体技术。
Mol Ther. 2020 Feb 5;28(2):411-421. doi: 10.1016/j.ymthe.2019.11.012. Epub 2019 Nov 16.
6
Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease.体内纵向监测中枢神经系统证明了基因治疗在 CLN5 神经鞘脂贮积病绵羊模型中的疗效。
Mol Ther. 2018 Oct 3;26(10):2366-2378. doi: 10.1016/j.ymthe.2018.07.015. Epub 2018 Jul 17.
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Prevention of Photoreceptor Cell Loss in a Cln6 Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells.预防感光细胞丧失在 CLN6 基因突变致神经鞘脂贮积病的鼠模型中需要 CLN6 基因转染双极细胞。
Mol Ther. 2018 May 2;26(5):1343-1353. doi: 10.1016/j.ymthe.2018.02.027. Epub 2018 Mar 2.
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Intravitreal implantation of TPP1-transduced stem cells delays retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis.玻璃体内植入转导TPP1的干细胞可延缓犬类CLN2神经元蜡样脂褐质沉积症的视网膜变性。
Exp Eye Res. 2016 Nov;152:77-87. doi: 10.1016/j.exer.2016.09.003. Epub 2016 Sep 13.
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AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.腺相关病毒基因转移可延缓晚期婴儿型神经元蜡样脂褐质沉积症（Batten病）TPP1缺陷犬模型的疾病发作。
Sci Transl Med. 2015 Nov 11;7(313):313ra180. doi: 10.1126/scitranslmed.aac6191.
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Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosis.酶替代疗法可延迟婴儿晚期神经元蜡样脂褐质沉积症犬模型的瞳孔光反射缺陷。
Exp Eye Res. 2014 Aug;125:164-72. doi: 10.1016/j.exer.2014.06.008. Epub 2014 Jun 19.