AIM: To develop a safe and efficient method for targeted, anti-apoptotic gene therapy of corneal endothelial cells (CECs). MATERIALS & METHODS: Magnetofection (MF), a combination of lipofection with magnetic nanoparticles (MNPs; PEI-Mag2, SO-Mag5, PalD1-Mag1), was tested in human CECs and in explanted human corneas. Effects on cell viability and function were investigated. Immunocompatibility was assessed in human peripheral blood mononuclear cells. RESULTS: Silica iron-oxide MNPs (SO-Mag5) combined with X-tremeGENE-HP achieved high transfection efficiency in human CECs and explanted human corneas, without altering cell viability or function. Magnetofection caused no immunomodulatory effects in human peripheral blood mononuclear cells. Magnetofection with anti-apoptotic P35 gene effectively blocked apoptosis in CECs. CONCLUSION: Magnetofection is a promising tool for gene therapy of corneal endothelial cells with potential for targeted on-site delivery.
AIM: To develop a safe and efficient method for targeted, anti-apoptotic gene therapy of corneal endothelial cells (CECs). MATERIALS & METHODS: Magnetofection (MF), a combination of lipofection with magnetic nanoparticles (MNPs; PEI-Mag2, SO-Mag5, PalD1-Mag1), was tested in human CECs and in explanted human corneas. Effects on cell viability and function were investigated. Immunocompatibility was assessed in human peripheral blood mononuclear cells. RESULTS:Silicairon-oxide MNPs (SO-Mag5) combined with X-tremeGENE-HP achieved high transfection efficiency in human CECs and explanted human corneas, without altering cell viability or function. Magnetofection caused no immunomodulatory effects in human peripheral blood mononuclear cells. Magnetofection with anti-apoptotic P35 gene effectively blocked apoptosis in CECs. CONCLUSION: Magnetofection is a promising tool for gene therapy of corneal endothelial cells with potential for targeted on-site delivery.
Entities:
Keywords:
apoptosis; corneal endothelium; gene therapy; magnetic nanoparticles; magnetofection
Authors: Philip Böhler; Fabian Stuhldreier; Ruchika Anand; Arun Kumar Kondadi; David Schlütermann; Niklas Berleth; Jana Deitersen; Nora Wallot-Hieke; Wenxian Wu; Marian Frank; Hendrik Niemann; Elisabeth Wesbuer; Andreas Barbian; Tomas Luyten; Jan B Parys; Stefanie Weidtkamp-Peters; Andrea Borchardt; Andreas S Reichert; Aida Peña-Blanco; Ana J García-Sáez; Samuel Itskanov; Alexander M van der Bliek; Peter Proksch; Sebastian Wesselborg; Björn Stork Journal: Cell Death Dis Date: 2018-02-19 Impact factor: 8.469