+34 620 10 75 37info@nanbiosis.com

Posts Taged cystic-fibrosis

You are here : Home / Posts Tagged "cystic fibrosis"

CRISPR technologies for Cystic Fibrosis treatment

Cystic fibrosis is an inherited genetic disease that mainly affects the lungs and the digestive system

On April 6, the first meeting was held, on the one hand, with people with Cystic Fibrosis and the Euskadi Cystic Fibrosis Association (Arnasa) and, on the other, with the NanoBioCel research group of the CIBER-BBN and the University of Basque Country, which is also part of the ICTS Nanbiosis through the Drug Formulation Unit U10.

The cooperative relationship between the NanoBioCel research group and Arnasa began approximately 5 years ago with the contributions made by the association to the NanBioCel group for research in cystic fibrosis.

The goup has focused on the development of non-viral vectors to address the treatment of cystic fibrosis through gene therapy. Progress is currently being made in the development of CRISPR technologies for its treatment.

This meeting was organized with the main objective of making those affected aware of the research carried out by NanoBioCel in cystic fibrosis and promoting associationism and collaboration between affected people and researchers to better understand the disease and find promising solutions.

The event was attended by almost 28 associates and many of the rest requested that the presentation of the researchers be recorded

Arnasa is the Cystic Fibrosis Association of the Basque Country and contributes with its work and activities to the promotion of general interest and the visibility of the disease, as well as to the improvement of the quality of life of people affected by the disease and their families.

In the picture: Increased activity of the chloride channel in CUFI cells transfected with non-viral vectors.

• Read More

1st Meeting with Cystic Fibrosis Patiens (NanoBioCel -ARNASA)

Cystic Fibrosis is a genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. It is a genetic, degenerative and chronic disease, with a population incidence is 1 in 5,000, so it is considered among the so called “Rare Diseases”

The most common symptoms are continuous colds, repeated infections, decreased lung capacity, impossibility of fat absorption and malnutrition

As there is no cure for the disease, daily treatment is the only option to have an acceptable quality of life

The NanoBioCell Research Group, which coordinates unit 10 of NANBIOSIS of the CIBER-BBN and the UPV/EHU, has a wide experience in comprehensive research into effective therapies for the treatment of cystic fibrosis and related diseases has organized the ‘First meeting with cystic fibrosis patients’ in the collaboration with ARNASA Bizkaia, (the Cystic Fibrosis Association of Bizkaia, made up of affected people, their families and volunteers who collaborate with the association)

Registration is free here. After registration you will receive a link to the online meeting

• Read More

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

• Read More