Gene Therapy Review: Gene and Cell Therapy Resource

Mice cured of lung cancer with gene therapy

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LONDON — Mice with up to 200 tumours have been completely cured of lung cancer using a “gene therapy” technique, scientists claim. By blocking production of a protein which drives the development of tumours, researchers were able to eliminate the tumours without any evidence of adverse sideeffects, the Daily Telegraph reported. The study found that the treatment was effective even after several rounds of therapy suggesting that the mice did not become resistant to it – a major problem for cancer doctors.

Scientists now hope to adapt the treatment into a form that is suitable and safe for use on humans so that its effectiveness can be tested in clinical trials. Dr Laura Soucek of the Vall d’Hebron Institute of Oncology in Barcelona, who led the study, said: “We’re so excited about reaching this turning point and I am quite certain that it will change the course of cancer therapy, despite there being a long road ahead.”

The therapy targeted a protein known as Myc, which plays an important role in our cells but can lead to uncontrolled cell growth and the onset of cancer if it is overproduced by the body. Mice were given an antibiotic in their drinking water which activated a mutant gene known as Omomyc, and this in turn blocked the production of Myc.

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Researchers show that lipid nanoparticles are ideal for delivering genes and drugs

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UPV/EHU-University of the Basque Country researchers are using nanotechnology for therapeutic purposes

This press release is available in Spanish.

		IMAGE: This shows the expression of the retinoschisis protein (green) after the transfection of a cell line of retina pigment epithelium (ARPE-19) with a lipid nanoparticle-based formulation with the RS1 gene.... Click here for more information.

At the Faculty of Pharmacy of the Basque Public University (UPV/EHU) the Pharmacokinetics, Nanotechnology and Gene Therapy research team is using nanotechnology to develop new formulations that can be applied to drugs and gene therapy.Specifically, they are using nanoparticles to designsystems for delivering genes and drugs; this helps to get the genes and drugs to the point of action so that they can produce the desired effect.

The research team has shown that lipid nanoparticles, which they have been working on for several years, are ideal for acting as vectors in gene therapy.Gene therapy is a highly promising alternative for diseases that so far have no effective treatment.It consists of delivering a nucleic acid, for example, a therapeutic gene, to modulate the expression of a protein that is found to be altered in a specific disease, thus reversing the biological disorder.

The main obstacle is that the genetic material cannot be formulated in conventional pharmaceutical ways, because it becomes degraded within the organism and cannot perform its function.To overcome this obstacle, viral vectors are normally used and they are able to deliver the therapeutic gene to the cells in which it has to act.However, as Dr Alicia Rodriguez explains, "viral vectors have a great drawback because they have a great potential to develop tumours.That is why there is a lot of interest in developing non-viral vectors, like vectors based on lipid nanoparticles."

Read more: Researchers show that lipid nanoparticles are ideal for delivering genes and drugs