The use of nanoparticles in gene therapy to treat rare diseases


Gene therapy is used to treat rare diseases like cystic fibrosis, diseases of the retina or the more common diseases such as AIDS, cancer and neurodegenerative disorders (Parkinson or Alzheimer).

The study by Ana Rodriguez del Pozo, a researcher at CIBER-BNN, Laboratory of Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy of the University of the Basque Country (UPV / EHU) is in the process of developing, characterize and optimize a system of administration of genes based on solid lipid nanoparticles (SLN). These particles, smaller than one micron are capable of delivering genes into cells and used to carry nucleic acids-DNA or RNA for use in gene therapies. These therapies could become effective therapeutic weapon against diseases that currently have that treatment palliatifs symptoms.

This research has also focused on factors related to the composition of nanoparticles that affects the transport capacity of genes to various cells, by studying the behavior of these particles inside the cells using various microscopy techniques. To optimize the action of the SLN, these are combined with a peptide which facilitates the entry of genes into cells. The peptides are organic compounds that are found in most living tissues and have multiple biological functions. On the other hand, this work has sought to improve the stability of nanoparticles, and therefore the SNL is to carry genes. For this, tests were conducted in vitro and latter through the intravenous administration in mice.

There are already therapies based on the administration of genes into cells, performed using a virus. These therapies are based on the natural ability of viruses to enter the nucleus of cells to apporter DNA or RNA. However, they may have an adverse effect on immunity. For this reason, it is interesting to develop particles with the same ability to penetrate cells but no effect on cellular immunity.

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