Until now, viral
systems have been the most effective method for delivering genetic
matter but they pose significant safety problems. Lipid nanoparticles (SLNs and NLCs) are regarded as highly promising
systems for delivering nucleic acids in gene therapy.
including SLNs and NLCs, are less effective but much safer even though
their effectiveness has increased significantly in recent years",
pointed out Alicia Rodríguez, María Ángeles Solinís and Ana del Pozo,
authors of the article published in the European Journal of Pharmaceutics and Biopharmaceutics
This review article describes these systems and their main
advantages in gene therapy, such as their capacity to protect the gene
material against degradation, to facilitate cell and nucleus
internalization and to boost the transfection process. "What is more,
the nanoparticles are made up of biocompatible, biodegradable materials,
they are easy to produce on a large scale, they can be sterilized and
freeze-dried and are very stable both in biological fluids and in
storage," explained the researchers.
This review also includes the main diseases in which lipid
nanoparticles are being applied, generally on the preclinical level:
degenerative diseases of the retina, infectious diseases, metabolic
disorders, and cancer, among others. "At PharmaNanoGene we are working
on the design and evaluation of SLNs for treating some of these diseases
using gene therapy. We are studying the relationship between
formulation factors and the processes involving the intracellular
internalisation and disposition of the genetic material that condition
the effectiveness of the vectors and which is essential in the
optimisation process, and for the first time we have demonstrated the
capacity of SLNs to induce the synthesis of a protein following their
intravenous administration in mice," they stressed.
The publication also includes other pieces of work by this UPV/EHU
research group on the application of SLNs in the treatment of rare
diseases, such as chromosome-X-linked juvenile retinoschisis, a disorder
in which the retina becomes destructured due to a deficiency in the
protein retinoschisin. "One of the main achievements of our studies in
this field has been to demonstrate, also for the first time, the
capacity of a non-viral vector to transfect the retina of animals
lacking the gene that encodes this protein and partially restore its
structure, showing than non-viral gene therapy is a viable, promising
therapeutic tool for treating degenerative disorders of the retina,"
specified the researchers.