A novel gene therapy for neurodegenerative Lafora disease via EPM2A-loaded DLinDMA lipoplexes
| Autor: | Vikas V. Dukhande, Aishwarya Saraswat, Shraddha Bhutkar, Ketan Patel, Hari Priya Vemana |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
0303 health sciences
Liposome Chemistry Genetic enhancement HEK 293 cells Biomedical Engineering Medicine (miscellaneous) Bioengineering Transfection Development Gene delivery medicine.disease Lafora disease 03 medical and health sciences 0302 clinical medicine Cancer research medicine General Materials Science Cationic liposome Laforin 030217 neurology & neurosurgery 030304 developmental biology |
| Zdroj: | Nanomedicine. 16:1081-1095 |
| ISSN: | 1748-6963 1743-5889 |
| DOI: | 10.2217/nnm-2020-0477 |
| Popis: | Aim: To develop novel cationic liposomes as a nonviral gene delivery vector for the treatment of rare diseases, such as Lafora disease – a neurodegenerative epilepsy. Materials & methods: DLinDMA and DOTAP liposomes were formulated and characterized for the delivery of gene encoding laforin and expression of functional protein in HEK293 and neuroblastoma cells. Results: Liposomes with cationic lipids DLinDMA and DOTAP showed good physicochemical characteristics. Nanosized DLinDMA liposomes demonstrated desired transfection efficiency, negligible hemolysis and minimal cytotoxicity. Western blotting confirmed successful expression and glucan phosphatase assay demonstrated the biological activity of laforin. Conclusion: Our study is a novel preclinical effort in formulating cationic lipoplexes containing plasmid DNA for the therapy of rare genetic diseases such as Lafora disease. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|