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| Item Type: | Article |
|---|---|
| Title: | Molecular and functional characterization of BDNF-overexpressing human retinal pigment epithelial cells established by sleeping beauty transposon-mediated gene transfer |
| Creators Name: | Mattern, L., Otten, K., Miskey, C., Fuest, M., Izsvák, Z., Ivics, Z., Walter, P., Thumann, G. and Johnen, S. |
| Abstract: | More and more patients suffer from multifactorial neurodegenerative diseases, such as age-related macular degeneration (AMD). However, their pathological mechanisms are still poorly understood, which complicates the development of effective therapies. To improve treatment of multifactorial diseases, cell-based gene therapy can be used to increase the expression of therapeutic factors. To date, there is no approved therapy for dry AMD, including late-stage geographic atrophy. We present a treatment option for dry AMD that transfers the brain-derived neurotrophic factor (BDNF) gene into retinal pigment epithelial (RPE) cells by electroporation using the plasmid-based Sleeping Beauty (SB) transposon system. ARPE-19 cells and primary human RPE cells were co-transfected with two plasmids encoding the (SB100X) transposase and the transposon carrying a BDNF transcription cassette. We demonstrated efficient expression and secretion of BDNF in both RPE cell types, which were further increased in ARPE-19 cell cultures exposed to hydrogen peroxide. BDNF-transfected cells exhibited lower apoptosis rates and stimulated neurite outgrowth in human SH-SY5Y cells. This study is an important step in the development of a cell-based BDNF gene therapy that could be applied as an advanced therapy medicinal product to treat dry AMD or other degenerative retinal diseases. |
| Keywords: | RPE Cells, BDNF, Sleeping Beauty Transposon System, Non-Viral Transfection, Cell-Based, Additive Gene Therapy, Neurodegenerative Retinal Diseases, AMD |
| Source: | International Journal of Molecular Sciences |
| ISSN: | 1422-0067 |
| Publisher: | MDPI |
| Volume: | 23 |
| Number: | 21 |
| Page Range: | 12982 |
| Date: | 1 November 2022 |
| Official Publication: | https://doi.org/10.3390/ijms232112982 |
| PubMed: | View item in PubMed |
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