视网膜基因治疗：21世纪的重大突破。 - Suppr

Suppr

超能文献

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

相似文献

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

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

Seeing the Light after 25 Years of Retinal Gene Therapy.

Trends Mol Med. 2018 Aug;24(8):669-681. doi: 10.1016/j.molmed.2018.06.006. Epub 2018 Jul 5.

Genetic Testing for Retina Specialists.

Ophthalmic Surg Lasers Imaging Retina. 2018 May 1;49(5):292-295. doi: 10.3928/23258160-20180501-01.

Laser-assisted targeted gene delivery to degenerated retina improves retinal function.

J Biophotonics. 2021 Jan;14(1):e202000234. doi: 10.1002/jbio.202000234. Epub 2020 Oct 19.

Gene supplementation therapy for recessive forms of inherited retinal dystrophies.

Gene Ther. 2012 Feb;19(2):154-61. doi: 10.1038/gt.2011.161. Epub 2011 Oct 27.

Genetic Rescue of X-Linked Retinoschisis Mouse () Retina Induces Quiescence of the Retinal Microglial Inflammatory State Following AAV8- Gene Transfer and Identifies Gene Networks Underlying Retinal Recovery.

Hum Gene Ther. 2021 Jul;32(13-14):667-681. doi: 10.1089/hum.2020.213. Epub 2020 Dec 14.

On Retinal Gene Therapy.

Ophthalmologica. 2016;236(1):1-7. doi: 10.1159/000445782. Epub 2016 Apr 28.

Inherited Eye Diseases with Retinal Manifestations through the Eyes of Homeobox Genes.

Int J Mol Sci. 2020 Feb 26;21(5):1602. doi: 10.3390/ijms21051602.

Prospects of Optogenetic Prosthesis of the Degenerative Retina of the Eye.

Biochemistry (Mosc). 2019 May;84(5):479-490. doi: 10.1134/S0006297919050031.

A Novel Method Combining Vitreous Aspiration and Intravitreal AAV2/8 Injection Results in Retina-Wide Transduction in Adult Mice.

Invest Ophthalmol Vis Sci. 2016 Oct 1;57(13):5326-5334. doi: 10.1167/iovs.16-19701.

引用本文的文献

Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia.

Brain Commun. 2025 Jan 30;7(1):fcaf046. doi: 10.1093/braincomms/fcaf046. eCollection 2025.

Expanding the Mutation Spectrum for Inherited Retinal Diseases.

Genes (Basel). 2024 Dec 28;16(1):32. doi: 10.3390/genes16010032.

Functional Roles of Pigment Epithelium-Derived Factor in Retinal Degenerative and Vascular Disorders: A Scoping Review.

Invest Ophthalmol Vis Sci. 2024 Dec 2;65(14):41. doi: 10.1167/iovs.65.14.41.

Subretinal AAV delivery of RNAi-therapeutics targeting reduces choroidal neovascularization in a large animal model.

Mol Ther Methods Clin Dev. 2024 Mar 22;32(2):101242. doi: 10.1016/j.omtm.2024.101242. eCollection 2024 Jun 13.

The RLR intrinsic antiviral system is expressed in neural retina and restricts lentiviral transduction of human Mueller cells.

Exp Eye Res. 2023 Nov;236:109647. doi: 10.1016/j.exer.2023.109647. Epub 2023 Sep 7.

Toward lentiviral vectors for antiangiogenic ocular gene therapy.

Mol Ther Methods Clin Dev. 2023 Aug 26;30:443-446. doi: 10.1016/j.omtm.2023.08.007. eCollection 2023 Sep 14.

The Bovine Ex Vivo Retina: A Versatile Model for Retinal Neuroscience.

Invest Ophthalmol Vis Sci. 2023 Aug 1;64(11):29. doi: 10.1167/iovs.64.11.29.

25 years of maturation: A systematic review of RNAi in the clinic.

Mol Ther Nucleic Acids. 2023 Jul 18;33:469-482. doi: 10.1016/j.omtn.2023.07.018. eCollection 2023 Sep 12.

MERTK missense variants in three patients with retinitis pigmentosa.

Ophthalmic Genet. 2023 Feb;44(1):74-82. doi: 10.1080/13816810.2022.2113541. Epub 2022 Aug 29.

Photodisruption of the Inner Limiting Membrane: Exploring ICG Loaded Nanoparticles as Photosensitizers.

Pharmaceutics. 2022 Aug 17;14(8):1716. doi: 10.3390/pharmaceutics14081716.

本文引用的文献

A single combination gene therapy treats multiple age-related diseases.

Proc Natl Acad Sci U S A. 2019 Nov 19;116(47):23505-23511. doi: 10.1073/pnas.1910073116. Epub 2019 Nov 4.

Safety of Same-Eye Subretinal Sequential Readministration of in Non-human Primates.

Mol Ther Methods Clin Dev. 2019 Sep 3;15:133-148. doi: 10.1016/j.omtm.2019.08.011. eCollection 2019 Dec 13.

Search-and-replace genome editing without double-strand breaks or donor DNA.

Nature. 2019 Dec;576(7785):149-157. doi: 10.1038/s41586-019-1711-4. Epub 2019 Oct 21.

Outcome Measures Used in Ocular Gene Therapy Trials: A Scoping Review of Current Practice.

Front Pharmacol. 2019 Sep 18;10:1076. doi: 10.3389/fphar.2019.01076. eCollection 2019.

Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning.

Nat Commun. 2019 Sep 19;10(1):4284. doi: 10.1038/s41467-019-12281-8.

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.

Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22.

Gene Therapy.

N Engl J Med. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910.

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration.

Gene Ther. 2019 Sep;26(9):386-398. doi: 10.1038/s41434-019-0094-3. Epub 2019 Jul 15.

Has retinal gene therapy come of age? From bench to bedside and back to bench.

Hum Mol Genet. 2019 Oct 1;28(R1):R108-R118. doi: 10.1093/hmg/ddz130.

Vascular dysfunction in retinitis pigmentosa.

Acta Ophthalmol. 2019 Nov;97(7):660-664. doi: 10.1111/aos.14138. Epub 2019 May 17.

文献AI研究员

20分钟写一篇综述，助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型，支持多种主流文档格式。

立即体验

相似文献

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

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

Seeing the Light after 25 Years of Retinal Gene Therapy.

Trends Mol Med. 2018 Aug;24(8):669-681. doi: 10.1016/j.molmed.2018.06.006. Epub 2018 Jul 5.

Genetic Testing for Retina Specialists.

Ophthalmic Surg Lasers Imaging Retina. 2018 May 1;49(5):292-295. doi: 10.3928/23258160-20180501-01.

Laser-assisted targeted gene delivery to degenerated retina improves retinal function.

J Biophotonics. 2021 Jan;14(1):e202000234. doi: 10.1002/jbio.202000234. Epub 2020 Oct 19.

Gene supplementation therapy for recessive forms of inherited retinal dystrophies.

Gene Ther. 2012 Feb;19(2):154-61. doi: 10.1038/gt.2011.161. Epub 2011 Oct 27.

Hum Gene Ther. 2021 Jul;32(13-14):667-681. doi: 10.1089/hum.2020.213. Epub 2020 Dec 14.

On Retinal Gene Therapy.

Ophthalmologica. 2016;236(1):1-7. doi: 10.1159/000445782. Epub 2016 Apr 28.

Inherited Eye Diseases with Retinal Manifestations through the Eyes of Homeobox Genes.

Int J Mol Sci. 2020 Feb 26;21(5):1602. doi: 10.3390/ijms21051602.

Prospects of Optogenetic Prosthesis of the Degenerative Retina of the Eye.

Biochemistry (Mosc). 2019 May;84(5):479-490. doi: 10.1134/S0006297919050031.

A Novel Method Combining Vitreous Aspiration and Intravitreal AAV2/8 Injection Results in Retina-Wide Transduction in Adult Mice.

Invest Ophthalmol Vis Sci. 2016 Oct 1;57(13):5326-5334. doi: 10.1167/iovs.16-19701.

引用本文的文献

Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia.

Brain Commun. 2025 Jan 30;7(1):fcaf046. doi: 10.1093/braincomms/fcaf046. eCollection 2025.

Expanding the Mutation Spectrum for Inherited Retinal Diseases.

Genes (Basel). 2024 Dec 28;16(1):32. doi: 10.3390/genes16010032.

Functional Roles of Pigment Epithelium-Derived Factor in Retinal Degenerative and Vascular Disorders: A Scoping Review.

Invest Ophthalmol Vis Sci. 2024 Dec 2;65(14):41. doi: 10.1167/iovs.65.14.41.

Subretinal AAV delivery of RNAi-therapeutics targeting reduces choroidal neovascularization in a large animal model.

Mol Ther Methods Clin Dev. 2024 Mar 22;32(2):101242. doi: 10.1016/j.omtm.2024.101242. eCollection 2024 Jun 13.

The RLR intrinsic antiviral system is expressed in neural retina and restricts lentiviral transduction of human Mueller cells.

Exp Eye Res. 2023 Nov;236:109647. doi: 10.1016/j.exer.2023.109647. Epub 2023 Sep 7.

Toward lentiviral vectors for antiangiogenic ocular gene therapy.

Mol Ther Methods Clin Dev. 2023 Aug 26;30:443-446. doi: 10.1016/j.omtm.2023.08.007. eCollection 2023 Sep 14.

The Bovine Ex Vivo Retina: A Versatile Model for Retinal Neuroscience.

Invest Ophthalmol Vis Sci. 2023 Aug 1;64(11):29. doi: 10.1167/iovs.64.11.29.

25 years of maturation: A systematic review of RNAi in the clinic.

Mol Ther Nucleic Acids. 2023 Jul 18;33:469-482. doi: 10.1016/j.omtn.2023.07.018. eCollection 2023 Sep 12.

MERTK missense variants in three patients with retinitis pigmentosa.

Ophthalmic Genet. 2023 Feb;44(1):74-82. doi: 10.1080/13816810.2022.2113541. Epub 2022 Aug 29.

Photodisruption of the Inner Limiting Membrane: Exploring ICG Loaded Nanoparticles as Photosensitizers.

Pharmaceutics. 2022 Aug 17;14(8):1716. doi: 10.3390/pharmaceutics14081716.

本文引用的文献

A single combination gene therapy treats multiple age-related diseases.

Proc Natl Acad Sci U S A. 2019 Nov 19;116(47):23505-23511. doi: 10.1073/pnas.1910073116. Epub 2019 Nov 4.

Safety of Same-Eye Subretinal Sequential Readministration of in Non-human Primates.

Mol Ther Methods Clin Dev. 2019 Sep 3;15:133-148. doi: 10.1016/j.omtm.2019.08.011. eCollection 2019 Dec 13.

Search-and-replace genome editing without double-strand breaks or donor DNA.

Nature. 2019 Dec;576(7785):149-157. doi: 10.1038/s41586-019-1711-4. Epub 2019 Oct 21.

Outcome Measures Used in Ocular Gene Therapy Trials: A Scoping Review of Current Practice.

Front Pharmacol. 2019 Sep 18;10:1076. doi: 10.3389/fphar.2019.01076. eCollection 2019.

Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning.

Nat Commun. 2019 Sep 19;10(1):4284. doi: 10.1038/s41467-019-12281-8.

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.

Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22.

Gene Therapy.

N Engl J Med. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910.

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration.

Gene Ther. 2019 Sep;26(9):386-398. doi: 10.1038/s41434-019-0094-3. Epub 2019 Jul 15.

Has retinal gene therapy come of age? From bench to bedside and back to bench.

Hum Mol Genet. 2019 Oct 1;28(R1):R108-R118. doi: 10.1093/hmg/ddz130.

Vascular dysfunction in retinitis pigmentosa.

Acta Ophthalmol. 2019 Nov;97(7):660-664. doi: 10.1111/aos.14138. Epub 2019 May 17.