% IMPORTANT: The following is UTF-8 encoded.  This means that in the presence
% of non-ASCII characters, it will not work with BibTeX 0.99 or older.
% Instead, you should use an up-to-date BibTeX implementation like “bibtex8” or
% “biber”.

@ARTICLE{Zhang:1044403,
      author       = {Zhang, Jiao and Bing, Ziyu and Marie, Corinne and Izsvák,
                      Zsuzsanna and Müller, Frank and Thumann, Gabriele and
                      Walter, Peter and Johnen, Sandra},
      title        = {{E}ffect of degeneration stage on non-viral tissue
                      transfection of rd10 retina ex vivo},
      journal      = {Molecular therapy / Nucleic Acids},
      volume       = {36},
      number       = {3},
      issn         = {2162-2531},
      address      = {New York, NY},
      publisher    = {Nature Publ. Group},
      reportid     = {FZJ-2025-03167},
      pages        = {102616 -},
      year         = {2025},
      note         = {This work was supported by the Confocal Microscope Facility
                      and the Immunohistochemistry Facility, core facilities of
                      the Interdisciplinary Center for Clinical Research (IZKF)
                      Aachen within the Faculty of Medicine at RWTH Aachen
                      University. Thethors thank Anne Freialdenhoven, Antje
                      Schiefer, and Alicia Hühnlein (Department of Ophthalmology,
                      University Hospital RWTH Aachen) for excellent
                      technicaltance. This work was funded by the China
                      Scholarship Council (no. 202208080251) and is part of a
                      graduate school funded by the Deutsche
                      Forschungsgemeinschaft DFG under GRK 2610/01. The results
                      shown here are part of the doctoral thesis of J.Z.},
      abstract     = {Gene therapy has great potential for the treatment of
                      inherited retinal diseases, as evidenced by the progress and
                      ongoing research. Using the Sleeping Beauty (SB) transposon
                      system, we developed a non-viral gene delivery system for
                      electroporation-based transfection of rd10 retinas ex vivo.
                      SB100X transposase and Venus transposon plasmids were
                      transfected at a ratio of 1:16 into rd10 retinas of
                      different ages and corresponding wild-type (WT) controls.
                      Transfection efficiency was assessed by fluorescence
                      microscopy and transfected cells were identified by
                      immunohistochemistry. Retinal integrity and cell viability
                      were assessed by FITC-dextran electroporation, histology,
                      and apoptosis assay. The highest transfection efficiency was
                      observed in degenerated stages P61 and older, with Müller
                      cells being the only transfected cell type. A $31\%$
                      reduction in transposon plasmid size resulted in a 1.5-fold
                      increase in transfection efficiency. Integrity and
                      morphology of degenerated retinas were preserved after
                      electroporation-based plasmid transfer; the number of
                      apoptotic cells in the inner nuclear layer (INL) was reduced
                      by half compared to WT controls. We demonstrated that
                      electroporation-based delivery of the SB transposon system
                      resulted in efficient transfection of degenerated retinas.
                      Our results are an important first step toward the combined
                      use of retinal prostheses and gene therapy to improve the
                      treatment of inherited retinal dystrophies.},
      cin          = {IBI-1 / INW-1},
      ddc          = {610},
      cid          = {I:(DE-Juel1)IBI-1-20200312 / I:(DE-Juel1)INW-1-20231219},
      pnm          = {5244 - Information Processing in Neuronal Networks
                      (POF4-524) / GRK 2610 - GRK 2610: Innovative Schnittstellen
                      zur Retina für optimiertes künstliches Sehen -
                      InnoRetVision (424556709)},
      pid          = {G:(DE-HGF)POF4-5244 / G:(GEPRIS)424556709},
      typ          = {PUB:(DE-HGF)16},
      doi          = {10.1016/j.omtn.2025.102616},
      url          = {https://juser.fz-juelich.de/record/1044403},
}