CRISPR-Cas9 could revolutionize the treatment of some cancers

Second the National Cancer Institute (Inca), 328,000 people are diagnosed with cancer every year in France. Of these people, nearly half will die from the disease. Cancer is a complex disease. Or rather we should talk about tumors. In fact, understand this type of file pathology is becoming clearer and in recent years we have realized that every tumor is different, especially within the cellular and protein changes it induces in the body.

Cancer treatments they are therefore generally heavy and long. Research is very active to find more effective and less harmful treatments for the patient’s body. In fact, even if the risk / benefit ratio of the treatments authorized to fight these diseases is positive, the treatments remain heavy to bear. Also, thanks toimmunotherapy.

Biotechnology could also help fight cancer: CRISPR-Cas9. This gene editing tool could, in theory, allow you to remodel the file genome cancer cells for, good, lead them to cell death (we speak ofapoptosis). However, the experiments carried out mainly reflect the difficulty in developing technologies so that Cas-9 shapes the genome as desired. This is the technical feat of Israeli researchers in a recent study Posted in Science.

A study with promising results

As mentioned above, exploit CRISPR-Cas9 biotechnology in the context of therapies against cancer was hampered by a low efficiency of the potential modeling genetics tumors and the potential toxicity Associates. Scientists behind the study, from the Precision and Nanomedicine Laboratory in Israel, created a nanoparticle lipid matrix to deliver Cas-9 safely to the body. To understand better, we interviewed Anna Gutkin, PhD student in nanomedicine, therapy through L ‘ARN, drug administration and co-author of the study.

Futura: How long have you been working with your team on this “lipid shuttle” development project to address the challenge of increasing publishing? gene through CRISPR-Cas-9 and reduced side effects? And how did the hypothesis come about?

Anna Gutkin: We have been working on this project for about five years. The hypothesis arose from the fact that the encapsulation ofMRNA Cas9 with sgRNA has been a major challenge in the research world due to the large size of the mRNA. To overcome this problem, we have developed a new class of lipids ionizable biodegradable (LIB) capable of encapsulating both mRNA and sgRNA. These lipids have an efficiency of transfection very high with very low toxicity (Ramishetti S et al. Adv. Mater. 2020). In addition, our LIBs are decorated withantibody targeting that improves cell specificity and reduces modification of normal viewers. Targeted antibodies are conjugated to LIBs using the ASSET binding system developed in our laboratory. This binding system allows us to easily change the target antibody and provide great versatility of target cells.

Futura: Did you encounter any difficulties or obstacles during your research? If so, which ones?

Anna Gutkin: The biggest challenge of this study was to develop an efficient and specific delivery vector for Cas9 and sgRNA mRNAs in a single vehicle. The relatively large size of Cas9 poses a major challenge for delivery into viral and non-viral delivery systems. We had to develop new lipids to enable this efficient release.

Futura: the path toapplication the clinical results are still long. How long do you think it will take?

Anna Gutkin: There is still a long way to go before this can be used in the clinic. However, we believe that with the development of vaccines at Moderna and Pfizer’s mRNA, the path to the clinic is getting shorter.