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Retinal gene therapy: an eye-opener of the 21st century.

作者信息

Askou Anne Louise, Jakobsen Thomas Stax, Corydon Thomas J

机构信息

Department of Biomedicine, Aarhus University, 8000, Aarhus C, Denmark.

Department of Ophthalmology, Aarhus University Hospital, 8200, Aarhus N, Denmark.

出版信息

Gene Ther. 2021 May;28(5):209-216. doi: 10.1038/s41434-020-0168-2. Epub 2020 Jun 19.

DOI:10.1038/s41434-020-0168-2
PMID:32561864
Abstract
摘要

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A single combination gene therapy treats multiple age-related diseases.一种单组合基因疗法可治疗多种与年龄相关的疾病。
Proc Natl Acad Sci U S A. 2019 Nov 19;116(47):23505-23511. doi: 10.1073/pnas.1910073116. Epub 2019 Nov 4.
2
Safety of Same-Eye Subretinal Sequential Readministration of in Non-human Primates.非人灵长类动物同眼视网膜下顺序重复给药的安全性。
Mol Ther Methods Clin Dev. 2019 Sep 3;15:133-148. doi: 10.1016/j.omtm.2019.08.011. eCollection 2019 Dec 13.
3
Search-and-replace genome editing without double-strand breaks or donor DNA.
色素上皮衍生因子在视网膜退行性和血管性疾病中的功能作用:一项范围综述
Invest Ophthalmol Vis Sci. 2024 Dec 2;65(14):41. doi: 10.1167/iovs.65.14.41.
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Subretinal AAV delivery of RNAi-therapeutics targeting reduces choroidal neovascularization in a large animal model.在大型动物模型中,通过视网膜下注射腺相关病毒(AAV)递送靶向RNA干扰疗法可减少脉络膜新生血管形成。
Mol Ther Methods Clin Dev. 2024 Mar 22;32(2):101242. doi: 10.1016/j.omtm.2024.101242. eCollection 2024 Jun 13.
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The RLR intrinsic antiviral system is expressed in neural retina and restricts lentiviral transduction of human Mueller cells.RLR 固有抗病毒系统在神经视网膜中表达,并限制人类 Muller 细胞慢病毒的转导。
Exp Eye Res. 2023 Nov;236:109647. doi: 10.1016/j.exer.2023.109647. Epub 2023 Sep 7.
6
Toward lentiviral vectors for antiangiogenic ocular gene therapy.用于抗血管生成眼部基因治疗的慢病毒载体研究进展
Mol Ther Methods Clin Dev. 2023 Aug 26;30:443-446. doi: 10.1016/j.omtm.2023.08.007. eCollection 2023 Sep 14.
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The Bovine Ex Vivo Retina: A Versatile Model for Retinal Neuroscience.牛离体视网膜:视网膜神经科学的多功能模型。
Invest Ophthalmol Vis Sci. 2023 Aug 1;64(11):29. doi: 10.1167/iovs.64.11.29.
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25 years of maturation: A systematic review of RNAi in the clinic.25年的发展历程:对临床RNA干扰技术的系统综述
Mol Ther Nucleic Acids. 2023 Jul 18;33:469-482. doi: 10.1016/j.omtn.2023.07.018. eCollection 2023 Sep 12.
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Pharmaceutics. 2022 Aug 17;14(8):1716. doi: 10.3390/pharmaceutics14081716.
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Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22.
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AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration.AAVrh-10 经玻璃体内给药后可转导啮齿动物和兔的外视网膜细胞。
Gene Ther. 2019 Sep;26(9):386-398. doi: 10.1038/s41434-019-0094-3. Epub 2019 Jul 15.
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Vascular dysfunction in retinitis pigmentosa.视网膜色素变性中的血管功能障碍。
Acta Ophthalmol. 2019 Nov;97(7):660-664. doi: 10.1111/aos.14138. Epub 2019 May 17.