Research progress in adeno-associated virus vectors for gene therapy of wet age-related macular degeneration_Chinese Journal of Ocular Fundus Diseases

Authors：

Li Fei ,  Xu Xinrong

Department of Ophthalmology, Affiliated Hospital of Nanjing University of Chinese Medicine, Nanjing 210029, China;

Corresponding?author：

Xu Xinrong, Email: xinrong_xu@aliyun.com

Keywords：

Age-related macular degeneration; Gene therapy; Adeno-associated virus; Viral vector; Review

DOI：

10.3760/cma.j.cn511434-20250226-00075

Video：

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Abstract Full text Figures/Tables Video References Cited by

Age-related macular degeneration (AMD) represents a significant cause of visual impairment and blindness in individuals over 65 years old. In recent years, gene therapy has emerged as a research hotspot for wet AMD, with adeno-associated virus (AAV) vectors being widely utilized due to their non-pathogenic nature, low immunogenicity, broad tissue tropism, and capacity for sustained transgene expression. Several related studies have progressed to clinical trial stages. Although challenges persist, including immunogenicity concerns, limited vector capacity, and potential long-term adverse effects, the continuous advancement of research strategies and technologies holds promise. Future developments may employ AAV delivery systems to achieve gene supplementation, gene editing, or gene silencing of angiogenesis-related signaling molecules, thereby providing novel therapeutic approaches for wet AMD.

Citation： Li Fei, Xu Xinrong. Research progress in adeno-associated virus vectors for gene therapy of wet age-related macular degeneration. Chinese Journal of Ocular Fundus Diseases, 2025, 41(9): 734-738. doi: 10.3760/cma.j.cn511434-20250226-00075 Copy

1.	Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis[J/OL]. Lancet Glob Health, 2014, 2(2): e106-e116[2014-01-03]. https://pubmed.ncbi.nlm.nih.gov/25104651/. DOI: 10.1016/S2214-109X(13)70145-1.
2.	李璐希, 張鵬. 新生血管性年齡相關性黃斑變性相關的視網膜下纖維化的研究進展[J]. 中華眼底病雜志, 2024, 40(3): 239-242. DOI: 10.3760/cma.j.cn511434-20230512-00221.Li LX, Zhang P. Research progress of subretinal fibrosis associated with neovascular age-related macular degeneration[J]. Chin J Ocul Fundus Dis, 2024, 40(3): 239-242. DOI: 10.3760/cma.j.cn511434-20230512-00221.
3.	Thomas CJ, Mirza RG, Gill MK. Age-related macular degeneration[J]. Med Clin North Am, 2021, 105(3): 473-491. DOI: 10.1016/j.mcna.2021.01.003.
4.	鄢聞嘉, 羅德倫, 馮加勁, 等. 眼底抗血管內皮生長因子藥物應用與創新[J]. 中華眼底病雜志, 2023, 39(8): 701-707. DOI: 10.3760/cma.j.cn511434-20220620-00371.Yan WJ, Luo DL, Feng JJ, et al. An update on anti-vascular endothelial growth factor therapy in retinal diseases[J]. Chin J Ocul Fundus Dis, 2023, 39(8): 701-707. DOI: 10.3760/cma.j.cn511434-20220620-00371.
5.	Chong V. Ranibizumab for the treatment of wet AMD: a summary of real-world studies[J]. Eye, 2016, 30(2): 270-286. DOI: 10.1038/eye.2015.217.
6.	Santiago-Ortiz JL, Schaffer DV. Adeno-associated virus (AAV) vectors in cancer gene therapy[J]. J Control Release, 2016, 240: 287-301. DOI: 10.1016/j.jconrel.2016.01.001.
7.	Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial[J]. Lancet, 2017, 390(10097): 849-860. DOI: 10.1016/S0140-6736(17)31868-8.
8.	Hendriks D, Clevers H, Artegiani B. CRISPR-Cas tools and their application in genetic engineering of human stem cells and organoids[J]. Cell Stem Cell, 2020, 27(5): 705-731. DOI: 10.1016/j.stem.2020.10.014.
9.	Zhou R, Caspi RR. Ocular immune privilege[J]. F1000 Biol Rep, 2010, 2: 3. DOI: 10.3410/B2-3.
10.	張燕宇, 高欣, 江寬, 等. 眼部疾病的基因治療與遞送策略[J]. 藥學學報, 2018, 53(4): 518-528. DOI: 10.16438/j.0513-4870.2017-0890.Zhang YY, Gao X, Jiang K, et al. Gene therapy and delivery strategies for ocular diseases[J]. Acta Pharmaceutica Sinica, 2018, 53(4): 518-528. DOI: 10.16438/j.0513-4870.2017-0890.
11.	陳永昌, 劉小平, 肖浦豪. 腺相關病毒載體及其在基因治療研究中的應用[J]. 昆明理工大學學報(自然科學版), 2024, 49(3): 188-201. DOI: 10.16112/j.cnki.53-1223/n.2024.03.661.Chen YC, Liu XP, Xiao PH. Adeno-associated virus vectors and their application in gene therapy research[J]. Journal of Kunming University of Science and Technology (Natural Science Edition), 2024, 49(3): 188-201. DOI: 10.16112/j.cnki.53-1223/n.2024.03.661.
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15.	Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery[J]. Nat Rev Drug Discov, 2019, 18(5): 358-378. DOI: 10.1038/s41573-019-0012-9.
16.	Penaud-Budloo M, Le Guiner C, Nowrouzi A, et al. Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle[J]. J Virol, 2008, 82(16): 7875-7885. DOI: 10.1128/JVI.00649-08.
17.	Ramlogan-Steel CA, Murali A, Andrzejewski S, et al. Gene therapy and the adeno-associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: trials, future directions and safety considerations[J]. Clin Exp Ophthalmol, 2019, 47(4): 521-536. DOI: 10.1111/ceo.13416.
18.	Daya S, Berns KI. Gene therapy using adeno-associated virus vectors[J]. Clin Microbiol Rev, 2008, 21(4): 583-593. DOI: 10.1128/CMR.00008-08.
19.	Tang JB, Wu YF, Cao Y, et al. Basic FGF or VEGF gene therapy corrects insufficiency in the intrinsic healing capacity of tendons[J/OL]. Sci Rep, 2016, 6: 20643[2016-02-11]. https://pubmed.ncbi.nlm.nih.gov/26865366/. DOI: 10.1038/srep20643.
20.	Barzel A, Paulk NK, Shi Y, et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice[J]. Nature, 2015, 517(7534): 360-364. DOI: 10.1038/nature13864.
21.	張敬法, 趙珍珍. 濕性年齡相關性黃斑變性發病機制及治療[J]. 眼科新進展, 2022, 42(2): 85-98. DOI: 10.13389/j.cnki.rao.2022.0019.Zhang JF, Zhao ZZ. Pathogenesis and treatment of wet age-related macular degeneration[J]. Rec Adv Ophthalmol, 2022, 42(2): 85-98. DOI: 10.13389/j.cnki.rao.2022.0019.
22.	Hussain RM, Ciulla TA. Emerging vascular endothelial growth factor antagonists to treat neovascular age-related macular degeneration[J]. Expert Opin Emerg Drugs, 2017, 22(3): 235-246. DOI: 10.1080/14728214.2017.1362390.
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24.	Heier JS, Kherani S, Desai S, et al. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial[J]. Lancet, 2017, 390(10089): 50-61. DOI: 10.1016/S0140-6736(17)30979-0.
25.	Constable IJ, Pierce CM, Lai CM, et al. Phase 2a randomized clinical trial: safety and post Hoc analysis of subretinal rAAV. sFLT-1 for wet age-related macular degeneration[J]. EBioMedicine, 2016, 14: 168-175. DOI: 10.1016/j.ebiom.2016.11.016.
26.	Kiss S, Oresic Bender K, Grishanin RN, et al. Long-term safety evaluation of continuous intraocular delivery of aflibercept by the intravitreal gene therapy candidate advm-022 in nonhuman primates[J]. Transl Vis Sci Technol, 2021, 10(1): 34. DOI: 10.1167/tvst.10.1.34.
27.	Khanani AM, Boyer DS, Wykoff CC, et al. Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study[J/OL]. EClinicalMedicine, 2024, 67: 102394[2023-12-22]. https://pubmed.ncbi.nlm.nih.gov/38152412/. DOI: 10.1016/j.eclinm.2023.102394.
28.	Campochiaro PA, Avery R, Brown DM, et al. Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study[J]. Lancet, 2024, 403(10436): 1563-1573. DOI: 10.1016/S0140-6736(24)00310-6.
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33.	陳有信, 湯加. 抗血管內皮生長因子治療眼部新生血管性疾病問題與挑戰[J]. 中華實驗眼科雜志, 2019, 37(1): 1-4. DOI: 10.3760/cma.j.issn.2095-0160.2019.01.001.Chen YX, Tang J. Problems and challenges of anti-vascular endothelial growth factor therapy for ocular neovescularization diseases[J]. Chin J Exp Ophthalmol, 2019, 37(1): 1-4. DOI: 10.3760/cma.j.issn.2095-0160.2019.01.001.
34.	Sadda SR, Guymer R, Monés JM, et al. Anti-vascular endothelial growth factor use and atrophy in neovascular age-related macular degeneration: systematic literature review and expert opinion[J. Ophthalmology, 2020, 127(5): 648-659. DOI: 10.1016/j.ophtha.2019.11.010.
35.	Davis JR, Banskota S, Levy JM, et al. Efficient prime editing in mouse brain, liver and heart with dual AAVs[J]. Nat Biotechnol, 2024, 42(2): 253-264. DOI: 10.1038/s41587-023-01758-z.
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1. Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis[J/OL]. Lancet Glob Health, 2014, 2(2): e106-e116[2014-01-03]. https://pubmed.ncbi.nlm.nih.gov/25104651/. DOI: 10.1016/S2214-109X(13)70145-1.
2. 李璐希, 張鵬. 新生血管性年齡相關性黃斑變性相關的視網膜下纖維化的研究進展[J]. 中華眼底病雜志, 2024, 40(3): 239-242. DOI: 10.3760/cma.j.cn511434-20230512-00221.Li LX, Zhang P. Research progress of subretinal fibrosis associated with neovascular age-related macular degeneration[J]. Chin J Ocul Fundus Dis, 2024, 40(3): 239-242. DOI: 10.3760/cma.j.cn511434-20230512-00221.
3. Thomas CJ, Mirza RG, Gill MK. Age-related macular degeneration[J]. Med Clin North Am, 2021, 105(3): 473-491. DOI: 10.1016/j.mcna.2021.01.003.
4. 鄢聞嘉, 羅德倫, 馮加勁, 等. 眼底抗血管內皮生長因子藥物應用與創新[J]. 中華眼底病雜志, 2023, 39(8): 701-707. DOI: 10.3760/cma.j.cn511434-20220620-00371.Yan WJ, Luo DL, Feng JJ, et al. An update on anti-vascular endothelial growth factor therapy in retinal diseases[J]. Chin J Ocul Fundus Dis, 2023, 39(8): 701-707. DOI: 10.3760/cma.j.cn511434-20220620-00371.
5. Chong V. Ranibizumab for the treatment of wet AMD: a summary of real-world studies[J]. Eye, 2016, 30(2): 270-286. DOI: 10.1038/eye.2015.217.
6. Santiago-Ortiz JL, Schaffer DV. Adeno-associated virus (AAV) vectors in cancer gene therapy[J]. J Control Release, 2016, 240: 287-301. DOI: 10.1016/j.jconrel.2016.01.001.
7. Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial[J]. Lancet, 2017, 390(10097): 849-860. DOI: 10.1016/S0140-6736(17)31868-8.
8. Hendriks D, Clevers H, Artegiani B. CRISPR-Cas tools and their application in genetic engineering of human stem cells and organoids[J]. Cell Stem Cell, 2020, 27(5): 705-731. DOI: 10.1016/j.stem.2020.10.014.
9. Zhou R, Caspi RR. Ocular immune privilege[J]. F1000 Biol Rep, 2010, 2: 3. DOI: 10.3410/B2-3.
10. 張燕宇, 高欣, 江寬, 等. 眼部疾病的基因治療與遞送策略[J]. 藥學學報, 2018, 53(4): 518-528. DOI: 10.16438/j.0513-4870.2017-0890.Zhang YY, Gao X, Jiang K, et al. Gene therapy and delivery strategies for ocular diseases[J]. Acta Pharmaceutica Sinica, 2018, 53(4): 518-528. DOI: 10.16438/j.0513-4870.2017-0890.
11. 陳永昌, 劉小平, 肖浦豪. 腺相關病毒載體及其在基因治療研究中的應用[J]. 昆明理工大學學報(自然科學版), 2024, 49(3): 188-201. DOI: 10.16112/j.cnki.53-1223/n.2024.03.661.Chen YC, Liu XP, Xiao PH. Adeno-associated virus vectors and their application in gene therapy research[J]. Journal of Kunming University of Science and Technology (Natural Science Edition), 2024, 49(3): 188-201. DOI: 10.16112/j.cnki.53-1223/n.2024.03.661.
12. Naso MF, Tomkowicz B, Perry WL 3rd, et al. Adeno-associated virus (AAV) as a vector for gene therapy[J]. Biodrugs, 2017, 31(4): 317-334. DOI: 10.1007/s40259-017-0234-5.
13. Greig JA, Martins KM, Breton C, et al. Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration[J]. Nat Biotechnol, 2024, 42(8): 1232-1242. DOI: 10.1038/s41587-023-01974-7.
14. Grieger JC, Choi VW, Samulski RJ. Production and characterization of adeno-associated viral vectors[J]. Nat Protoc, 2006, 1(3): 1412-1428. DOI: 10.1038/nprot.2006.207.
15. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery[J]. Nat Rev Drug Discov, 2019, 18(5): 358-378. DOI: 10.1038/s41573-019-0012-9.
16. Penaud-Budloo M, Le Guiner C, Nowrouzi A, et al. Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle[J]. J Virol, 2008, 82(16): 7875-7885. DOI: 10.1128/JVI.00649-08.
17. Ramlogan-Steel CA, Murali A, Andrzejewski S, et al. Gene therapy and the adeno-associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: trials, future directions and safety considerations[J]. Clin Exp Ophthalmol, 2019, 47(4): 521-536. DOI: 10.1111/ceo.13416.
18. Daya S, Berns KI. Gene therapy using adeno-associated virus vectors[J]. Clin Microbiol Rev, 2008, 21(4): 583-593. DOI: 10.1128/CMR.00008-08.
19. Tang JB, Wu YF, Cao Y, et al. Basic FGF or VEGF gene therapy corrects insufficiency in the intrinsic healing capacity of tendons[J/OL]. Sci Rep, 2016, 6: 20643[2016-02-11]. https://pubmed.ncbi.nlm.nih.gov/26865366/. DOI: 10.1038/srep20643.
20. Barzel A, Paulk NK, Shi Y, et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice[J]. Nature, 2015, 517(7534): 360-364. DOI: 10.1038/nature13864.
21. 張敬法, 趙珍珍. 濕性年齡相關性黃斑變性發病機制及治療[J]. 眼科新進展, 2022, 42(2): 85-98. DOI: 10.13389/j.cnki.rao.2022.0019.Zhang JF, Zhao ZZ. Pathogenesis and treatment of wet age-related macular degeneration[J]. Rec Adv Ophthalmol, 2022, 42(2): 85-98. DOI: 10.13389/j.cnki.rao.2022.0019.
22. Hussain RM, Ciulla TA. Emerging vascular endothelial growth factor antagonists to treat neovascular age-related macular degeneration[J]. Expert Opin Emerg Drugs, 2017, 22(3): 235-246. DOI: 10.1080/14728214.2017.1362390.
23. 袁運. 重組腺相關病毒基因治療年齡相關性黃斑變性的研究[D]. 合肥: 中國科學技術大學, 2024.Yuan Y. Research on recombinant adeno-associated virus gene therapy for age-related macular degeneration[D]. Hefei: University of Science and Technology of China, 2024.
24. Heier JS, Kherani S, Desai S, et al. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial[J]. Lancet, 2017, 390(10089): 50-61. DOI: 10.1016/S0140-6736(17)30979-0.
25. Constable IJ, Pierce CM, Lai CM, et al. Phase 2a randomized clinical trial: safety and post Hoc analysis of subretinal rAAV. sFLT-1 for wet age-related macular degeneration[J]. EBioMedicine, 2016, 14: 168-175. DOI: 10.1016/j.ebiom.2016.11.016.
26. Kiss S, Oresic Bender K, Grishanin RN, et al. Long-term safety evaluation of continuous intraocular delivery of aflibercept by the intravitreal gene therapy candidate advm-022 in nonhuman primates[J]. Transl Vis Sci Technol, 2021, 10(1): 34. DOI: 10.1167/tvst.10.1.34.
27. Khanani AM, Boyer DS, Wykoff CC, et al. Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study[J/OL]. EClinicalMedicine, 2024, 67: 102394[2023-12-22]. https://pubmed.ncbi.nlm.nih.gov/38152412/. DOI: 10.1016/j.eclinm.2023.102394.
28. Campochiaro PA, Avery R, Brown DM, et al. Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study[J]. Lancet, 2024, 403(10436): 1563-1573. DOI: 10.1016/S0140-6736(24)00310-6.
29. Chung SH, Sin TN, Ngo T, et al. CRISPR technology for ocular angiogenesis[J/OL]. Front Genome Ed, 2020, 2: 594984[2020-12-22]. https://pubmed.ncbi.nlm.nih.gov/34713223/. DOI: 10.3389/fgeed.2020.594984.
30. Sundaresan Y, Yacoub S, Kodati B, et al. Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases[J]. FEBS J, 2023, 290(22): 5248-5269. DOI: 10.1111/febs.16771.
31. Kim E, Koo T, Park SW, et al. In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni[J/OL]. Nat Commun, 2017, 8: 14500[2017-02-21]. https://pubmed.ncbi.nlm.nih.gov/28220790/. DOI: 10.1038/ncomms14500.
32. 秦勛, 劉張愉, 黃嘉鈺, 等. 基因補充與CRISPR/Cas9基因編輯在新生血管性眼底病中的應用[J]. 眼科新進展, 2025, 45(3): 233-237. DOI: 10.13389/j.cnki.rao.2025.0041.Qin X, Liu ZY, Huang JY, et al. The application of gene augmentation and CRISPR/Cas9 gene editing to the treatment of neovascular fundus diseases[J]. Rec Adv Ophthalmol, 2025, 45(3): 233-237. DOI: 10.13389/j.cnki.rao.2025.0041.
33. 陳有信, 湯加. 抗血管內皮生長因子治療眼部新生血管性疾病問題與挑戰[J]. 中華實驗眼科雜志, 2019, 37(1): 1-4. DOI: 10.3760/cma.j.issn.2095-0160.2019.01.001.Chen YX, Tang J. Problems and challenges of anti-vascular endothelial growth factor therapy for ocular neovescularization diseases[J]. Chin J Exp Ophthalmol, 2019, 37(1): 1-4. DOI: 10.3760/cma.j.issn.2095-0160.2019.01.001.
34. Sadda SR, Guymer R, Monés JM, et al. Anti-vascular endothelial growth factor use and atrophy in neovascular age-related macular degeneration: systematic literature review and expert opinion[J. Ophthalmology, 2020, 127(5): 648-659. DOI: 10.1016/j.ophtha.2019.11.010.
35. Davis JR, Banskota S, Levy JM, et al. Efficient prime editing in mouse brain, liver and heart with dual AAVs[J]. Nat Biotechnol, 2024, 42(2): 253-264. DOI: 10.1038/s41587-023-01758-z.
36. Guo C, Ma X, Gao F, et al. Off-target effects in CRISPR/Cas9 gene editing[J/OL]. Front Bioeng Biotechnol, 2023, 11: 1143157[2023-03-23]. https://pubmed.ncbi.nlm.nih.gov/36970624/. DOI: 10.3389/fbioe.2023.1143157.
37. 萬博, 金子兵. 腺相關病毒不同遞送途徑對視網膜基因治療影響的研究進展[J]. 中華眼底病雜志, 2024, 40(5): 409-414. DOI: 10.3760/cma.j.cn511434-20230918-00391.Wan B, Jin ZB. Research progress of effect of different delivery routes of adeno-associated virus on retinal gene therapy[J]. Chin J Ocul Fundus Dis, 2024, 40(5): 409-414. DOI: 10.3760/cma.j.cn511434-20230918-00391.
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Research progress in adeno-associated virus vectors for gene therapy of wet age-related macular degeneration

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