305
Views
4
CrossRef citations to date
0
Altmetric
Retina

A rAAV2/6 Mutant with Enhanced Targeting for Mouse Retinal Müller Cells

, , , , , , & show all
Pages 64-71 | Received 25 Mar 2019, Accepted 26 Jun 2019, Published online: 27 Aug 2019

References

  • Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev. 2008 Oct;21(4):583–93. doi:10.1128/CMR.00008-08.
  • Santiago-Ortiz JL, Schaffer DV. Adeno-associated virus (AAV) vectors in cancer gene therapy. J Control Release. 2016 Oct 28;240:287–301. doi:10.1016/j.jconrel.2016.01.001.
  • Xie Y, Hicks MJ, Kaminsky SM, Moore MA, Crystal RG, Rafii A. AAV-mediated persistent bevacizumab therapy suppresses tumor growth of ovarian cancer. Gynecol Oncol. 2014 Nov;135(2):325–32. doi:10.1016/j.ygyno.2014.07.105.
  • Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011 Dec 22;365(25):2357–65. doi:10.1056/NEJMoa1108046.
  • Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY et al. AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017 Dec 28;377(26):2519–30. doi:10.1056/NEJMoa1708483.
  • Ciesielska A, Samaranch L, San Sebastian W, Dickson DW, Goldman S, Forsayeth J, Bankiewicz KS. Depletion of AADC activity in caudate nucleus and putamen of Parkinson’s disease patients; implications for ongoing AAV2-AADC gene therapy trial. PLoS One. 2017 Feb 6;12(2):e0169965. doi:10.1371/journal.pone.0169965.
  • Faustini G, Longhena F, Varanita T, Bubacco L, Pizzi M, Missale C, Benfenati F, Bjorklund A, Spano P, Bellucci A. Synapsin III deficiency hampers alpha-synuclein aggregation, striatal synaptic damage and nigral cell loss in an AAV-based mouse model of Parkinson’s disease. Acta Neuropathol. 2018 Oct;136(4):621–39. doi:10.1007/s00401-018-1892-1.
  • Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr., Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM et al. Safety and efficacy of gene transfer for Leber’s congenital amaurosis. N Engl J Med. 2008 May 22;358(21):2240–48. doi:10.1056/NEJMoa0802315.
  • Smalley E. First AAV gene therapy poised for landmark approval. Nat Biotechnol. 2017 Nov 9;35(11):998–99. doi:10.1038/nbt1117-998.
  • Heier JS, Kherani S, Desai S, Dugel P, Kaushal S, Cheng SH, Delacono C, Purvis A, Richards S, Le-Halpere A et al. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial. Lancet. 2017 Jul 1;390(10089):50–61. doi:10.1016/S0140-6736(17)30979-0.
  • Constable IJ, Lai CM, Magno AL, French MA, Barone SB, Schwartz SD, Blumenkranz MS, Degli-Esposti MA, Rakoczy EP. Gene therapy in neovascular age-related macular degeneration: three-year follow-up of a Phase 1 randomized dose escalation trial. Am J Ophthalmol. 2017 May;177:150–58. doi:10.1016/j.ajo.2017.02.018.
  • Ghazi NG, Abboud EB, Nowilaty SR, Alkuraya H, Alhommadi A, Cai H, Hou R, Deng WT, Boye SL, Almaghamsi A, et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial. Hum Genet. 2016 Mar;135(3):327–43. doi:10.1007/s00439-016-1637-y.
  • Beltran WA, Cideciyan AV, Boye SE, Ye GJ, Iwabe S, Dufour VL, Marinho LF, Swider M, Kosyk MS, Sha J et al. Optimization of retinal gene therapy for X-linked retinitis pigmentosa due to RPGR mutations. Mol Ther. 2017 Aug 2;25(8):1866–80. doi:10.1016/j.ymthe.2017.05.004.
  • Edwards TL, Jolly JK, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Black GC, Webster AR, Lotery AJ, Holder GE et al. Visual acuity after retinal gene therapy for choroideremia. N Engl J Med. 2016 May 19;374(20):1996–98. doi:10.1056/NEJMc1509501.
  • Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, Tanaka JC, Acland GM, Hauswirth WW, Aguirre GD. Gene therapy rescues cone function in congenital achromatopsia. Hum Mol Genet. 2010 Jul 1;19(13):2581–93. doi:10.1093/hmg/ddq136.
  • Byrne LC, Oztürk BE, Lee T, Fortuny C, Visel M, Dalkara D, Schaffer DV, Flannery JG. Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h-/- mouse. Gene Ther. 2014 Jun;21(6):585–92. doi:10.1038/gt.2014.31.
  • Devoldere J, Peynshaert K, De Smedt SC, Remaut K. Müller cells as a target for retinal therapy. Drug Discov Today. 2019 Feb 4. pii ;S1359–6446(18):30431–38. doi:10.1016/j.drudis.2019.01.023.
  • Biedermann B, Bringmann A, Reichenbach A. High-affinity GABA uptake in retinal glial (Müller) cells of the guinea pig: electrophysiological characterization, immunohistochemical localization, and modeling of efficiency. Glia. 2002 Sep;39(3):217–28. doi:10.1002/glia.10097.
  • Winkler BS, Arnold MJ, Brassell MA, Puro DG. Energy metabolism in human retinal Müller cells. Invest Ophthalmol Vis Sci. 2000 Sep;41(10):3183–90.
  • Abukawa H, Tomi M, Kiyokawa J, Hori S, Kondo T, Terasaki T, Hosoya K. Modulation of retinal capillary endothelial cells by Müller glial cell-derived factors. Mol Vis. 2009;15:451–57.
  • Pellissier LP, Hoek RM, Vos RM, Aartsen WM, Klimczak RR, Hoyng SA, Flannery JG, Wijnholds J. Specific tools for targeting and expression in Müller glial cells. Mol Ther Methods Clin Dev. 2014 Mar 19;1:14009. doi:10.1038/mtm.2014.9.
  • Koerber JT, Klimczak R, Jang JH, Dalkara D, Flannery JG, Schaffer DV. Molecular evolution of adeno-associated virus for enhanced glial gene delivery. Mol Ther. 2009 Dec;17(12):2088–95. doi:10.1038/mt.2009.184.
  • Girod A, Ried M, Wobus C, Lahm H, Leike K, Kleinschmidt J, Deleage G, Hallek M. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat Med. 1999 Sep 5;(9):1052–56. doi:10.1038/12491.
  • Buning H, Ried MU, Perabo L, Gerner FM, Huttner NA, Enssle J, Hallek M. Receptor targeting of adeno-associated virus vectors. Gene Ther. 2003 Jul;10(14):1142–51. doi:10.1038/sj.gt.3301976.
  • Hickey DG, Edwards TL, Barnard AR, Singh MS, de Silva SR, McClements ME, Flannery JG, Hankins MW, MacLaren RE. Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina. Gene Ther. 2017 Dec;24(12):787–800. doi:10.1038/gt.2017.85.
  • Klimczak RR, Koerber JT, Dalkara D, Flannery JG, Schaffer DV. A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PLoS One. 2009 Oct 14;4(10):e7467. doi:10.1371/journal.pone.0007467.
  • Wu Z, Miller E, Agbandje-McKenna M, Samulski RJ. Alpha2,3 and alpha2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6. J Virol. 2006 Sep;80(18):9093–103. doi:10.1128/JVI.00895-06.
  • Akache B, Grimm D, Pandey K, Yant SR, Xu H, Kay MA. The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol. 2006 Oct;80(19):9831–36. doi:10.1128/JVI.00878-06.
  • Byrne LC, Khalid F, Lee T, Zin EA, Greenberg KP, Visel M, Schaffer DV, Flannery JG. AAV-mediated, optogenetic ablation of Müller Glia leads to structural and functional changes in the mouse retina. PLoS One. 2013 Sep 27;8(9):e76075. doi:10.1371/journal.pone.0076075.
  • Aartsen WM, van Cleef KW, Pellissier LP, Hoek RM, Vos RM, Blits B, Ehlert EM, Balaggan KS, Ali RR, Verhaagen J et al. GFAP-driven GFP expression in activated mouse Müller glial cells aligning retinal blood vessels following intravitreal injection of AAV2/6 vectors. PLoS One. 2010 Aug 24;5(8):e12387. doi:10.1371/journal.pone.0012387.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.