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Posts Taged non-viral-vectors-for-gene-therapy

Non-viral mediated gene therapy in human cystic fibrosis airway epithelial cells recovers chloride channel functionality

Researchers of CIBER-BBN Units of NANBIOSIS: U29 Oligonucleotide Synthesis Platform (OSP) at IQAC_CSIC, led by Prof. Ramón Eritja and U10 Drug Formulation, at UPV-EHU, led by Prof José Luis Pedraz, are coauthors of an article published by International Journal of Pharmaceutics.

Gene therapy strategies based on non-viral vectors are currently considered as a promising therapeutic option for the treatment of cystic fibrosis (CF), being liposomes the most commonly used gene carriers. Niosomes offer a powerful alternative to liposomes due to their higher stability and lower cytotoxicity, provided by their non-ionic surfactant and helper components. In this work, a three-formulation screening is performed, in terms of physicochemical and biological behavior, in CF patient derived airway epithelial cells. The most efficient niosome formulation reaches 28% of EGFP expressing live cells and follows caveolae-mediated endocytosis. Transfection with therapeutic cystic fibrosis transmembrane conductance regulator (CFTR) gene results in 5-fold increase of CFTR protein expression in transfected versus non-transfected cells, which leads to 1.5-fold increment of the chloride channel functionality. These findings highlight the relevance of niosome-based systems as an encouraging non-viral gene therapy platform with potential therapeutic benefits for CF.

The article acknowledges U10 Drug Formulation, for the intellectual and technical assistance

Article or reference:

Non-viral mediated gene therapy in human cystic fibrosis airway epithelial cells recovers chloride channel functionality-Sainz-Ramos, M., Villate-Beitia, I., Gallego, I., A.L. Qtaish, N., Lopez-Mendez, T.B., Eritja, R., Grijalvo, S., Puras, G., Pedraz, J.L. International Journal of Pharmaceutics, 588, art. no. 119757, 2020. https://doi.org/10.1016/j.ijpharm.2020.119757

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I Forum on Emerging Technologies organized by CIBER

On May 8th, will take place in Madrid, at the Assembly Hall Ernest Lluch of the Instituto de Salud Carlos III, organized by CIBER Internationalization Platform (CIBER-BBN, CIBERER, CIBERES) the I Forum on Emerging Technologies.

The objective of this meeting is to promote the exchange of ideas and scientific knowledge among the CIBER research groups with the aim of generating new collaborations for participation in cross-cutting projects and the development of border technologies. The content of the forum, centered on the type of technology and possibilities it offers, is structured in a program of lectures and scientific debates whose final objective is the discussion and the joint generation of ideas. This first edition of the forum will be focused on Gene Therapy, Nanotechnologies and Omic Technologies-Big Data.

Some Units of NANBIOSIS will participate:

  • José Luis Pedraz, Scientific Director of  Unit 10 Drug Formulation, will speak of “Development of non-viral vectors for gene therapy
  • Laura Lechuga, Scientific Director of Unit 4  Biodepositon and Biodetection, will talk about “Nanodispositive biosensors for advanced clinical diagnosis
  • Rosa Villa, Scientific Director of Unit 8 Micro–Nano Technology, Ramón Martínez, Scientific Director of Unit 26 NMR Biomedical Applications II and Esther Pueyo, researcher of U27 High Performance Computer will moderate the sessions.

 

Programme and registration details

 

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