Progress on ocular siRNA gene-silencing therapy and drug delivery systems

被引：36
作者：
Jiang, Jinjin [1 ,2 ]
Zhang, Xinru [1 ,2 ]
Tang, Yue [1 ,2 ]
Li, Shuhan [2 ]
Chen, Jing [2 ]
机构：
[1] Guizhou Med Univ, State Key Lab Funct & Applicat Med Plants, 1 DongQing Rd, Guiyang 550014, Peoples R China
[2] China Pharmaceut Univ, Dept Pharm, 639 Longmian Ave, Nanjing 211198, Peoples R China
来源：
FUNDAMENTAL & CLINICAL PHARMACOLOGY | 2021年 / 35卷 / 01期
关键词：
age-related macular degeneration; glaucoma; RNA interference technology; siRNA drugs; non-viral vectors; ENDOTHELIAL GROWTH-FACTOR; BETA-ADRENERGIC RECEPTORS; VEGF SIRNA; POSTERIOR SEGMENT; POLYETHYLENIMINE NANOPARTICLES; INTRAVITREAL INJECTION; MACULAR DEGENERATION; NONVIRAL VECTORS; RNA INTERFERENCE; CLINICAL-TRIAL;
D O I：
10.1111/fcp.12561
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
Age-related macular degeneration (AMD) and glaucoma are global ocular diseases with high blindness rate. RNA interference (RNAi) is being increasingly used in the treatment of these disorders with siRNA drugs, bevasiranib, AGN211745 and PF-04523655 for AMD, and SYL040012 and QPI-1007 for glaucoma. Administration routes and vectors of gene drugs affect their therapeutic effect. Compared with the non-viral vectors, viral vectors have limited payload capacity and potential immunogenicity. This review summarizes the progress of the ocular siRNA gene-silencing therapy by focusing on siRNA drugs for AMD and glaucoma already used in clinical research, the main routes of drug delivery and the non-viral vectors for siRNA drugs.
引用
收藏
页码：4 / 24
页数：21
相关论文
共 127 条
  • [81] Nanoparticle Delivery Enhancement With Acoustically Activated Microbubbles
    Mullin, Lee B.
    Phillips, Linsey C.
    Dayton, Paul A.
    [J]. IEEE TRANSACTIONS ON ULTRASONICS FERROELECTRICS AND FREQUENCY CONTROL, 2013, 60 (01) : 65 - 77
  • [82] Anti-tumor effects of anti-VEGF siRNA encapsulated with PLGA microspheres in mice
    Murata, Naoyuki
    Takashima, Yuuki
    Toyoshima, Kosei
    Yamamoto, Mari
    Okada, Hiroaki
    [J]. JOURNAL OF CONTROLLED RELEASE, 2008, 126 (03) : 246 - 254
  • [83] Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients
    Nguyen, Q. D.
    Schachar, R. A.
    Nduaka, C. I.
    Sperling, M.
    Basile, A. S.
    Klamerus, K. J.
    Chi-Burris, K.
    Yan, E.
    Paggiarino, D. A.
    Rosenblatt, I.
    Khan, A.
    Aitchison, R.
    Erlich, S. S.
    [J]. EYE, 2012, 26 (08) : 1099 - 1105
  • [84] Polyethylenimine nanoparticles as an efficient in vitro siRNA delivery system
    Nimesh, Surendra
    Chandra, Ramesh
    [J]. EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS, 2009, 73 (01) : 43 - 49
  • [85] Non-viral strategies for ocular gene delivery
    Oliveira, Ana V.
    da Costa, Ana M. Rosa
    Silva, Gabriela A.
    [J]. MATERIALS SCIENCE & ENGINEERING C-MATERIALS FOR BIOLOGICAL APPLICATIONS, 2017, 77 : 1275 - 1289
  • [86] Current status of polymeric gene delivery systems
    Park, Tae Gwan
    Jeong, Ji Hoon
    Kim, Sung Wan
    [J]. ADVANCED DRUG DELIVERY REVIEWS, 2006, 58 (04) : 467 - 486
  • [87] Vitreous: A barrier to nonviral ocular gene therapy
    Peeters, L
    Sanders, NN
    Braeckmans, K
    Boussery, K
    Van de Voorde, J
    De Smedt, SC
    Demeester, J
    [J]. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2005, 46 (10) : 3553 - 3561
  • [88] Influence of Disulfide Density and Molecular Weight on Disulfide Cross-Linked Polyethylenimine as Gene Vectors
    Peng, Qi
    Hu, Chu
    Cheng, Juan
    Zhong, Zhenlin
    Zhuo, Renxi
    [J]. BIOCONJUGATE CHEMISTRY, 2009, 20 (02) : 340 - 346
  • [89] Subretinal Injection: A Review on the Novel Route of Therapeutic Delivery for Vitreoretinal Diseases
    Peng, Yingqian
    Tang, Luosheng
    Zhou, Yedi
    [J]. OPHTHALMIC RESEARCH, 2017, 58 (04) : 217 - 226
  • [90] Morphology and Composition of the Inner Limiting Membrane: Species-Specific Variations and Relevance toward Drug Delivery Research
    Peynshaert, Karen
    Devoldere, Joke
    Minnaert, An-Katrien
    De Smedt, Stefaan C.
    Remaut, Katrien
    [J]. CURRENT EYE RESEARCH, 2019, 44 (05) : 465 - 475
45678910111213