Gene Editing to Cure

Strengthening Current methods to treat non-communicable diseases through innovative technologies of genome editing

Traditional practices and methods used to treat many non-communicable diseases are not sufficient to tackle their increasing burden throughout the world. It is becoming clear that new developments and approaches in health research can offer innovative and efficient solutions to these problems. Through the cooperation and training spearheaded by the ICGEB, innovative therapies and the means to develop them are now becoming available in low-resource settings.  

Gene editing is emerging as one of the key new technologies in this arena. It is a technology that allows the genetic information of mammalian cells to be edited with great precision, thus potentially allowing the correction of mutations linked to human diseases. It is a relatively easy to implement and inexpensive technology that can be implemented in any molecular biology laboratory. 

Since 2016, the ICGEB has been instrumental in creating South-South platforms to share knowledge on the most modern technologies in gene editing and their application in the medical field through the organization of theoretical and practical workshops. The areas covered by these workshops are:  

• the generation of animal models carrying the mutations associated with human diseases in order to gain insights into the molecular mechanisms responsible for disease onset and progression; 
• the introduction of these mutations into embryonic stem cells and induced pluripotent stem (iPS) cells in order to create cellular models of the disease and use them to screen for the efficacy of particular drugs, or to understand the inter-individual variability in the manifestations of a disease caused by the same genetic mutation (precision medicine);  
• the development of innovative therapies based on gene editing. 

This technology can be applied to the development of simple, cost-effective models in such organisms as zebrafish and C. elegans worms, which can be used to study the implications of gene modification in non-communicable diseases. This leads to more impactful science and thus an improved ability to apply for research funding; more importantly, it leads to results that are more likely to be translated into applicable therapeutics. 

These ICGEB international workshops allow scientists from Member States to understand the most recent gene editing procedures and to apply them in their own countries and laboratories. During these workshops, scientists from the ICGEB constituency learn from each other, share technology, start new collaborations, and have the opportunity to discuss the ethical and regulatory issues associated with the technology. One tangible example is the ‘ICGEB – Joint Research Centre (JRC) Workshop on Genome Editing’, which gathered expertise from Belgium, Italy, Kenya, Latvia, Nigeria, Thailand and the United Kingdom, , and strengthened collaboration among various academic institutions and laboratories in an effort to provide consultancy on the regulatory, ethical and societal challenges driven by the technology and its applications. South-South and triangular cooperation are nurtured also via technology transfer and collaborations with the industry in Argentina, Brazil, Egypt, Iran, Lebanon, Syrian Arab Republic, among others.  

International workshops represent a unique opportunity to gain the knowledge necessary to extend the impact of correlation studies on the gene expression, which are increasingly being performed in the Global South, as documented by the numbers of grant applications on this subject that have been received by the ICGEB in the current year.  

The outcome of the practice is ultimately capacity building and knowledge transfer in the Global South, which goes beyond the traditional practices and methods used to treat non-communicable diseases and to the vanguard field of gene editing. 

Being directly linked with the aim of reducing mortality rates of non-communicable diseases, gene editing contributes to improving treatment plans and enriching genetic information about the onset and progression of diseases.   

The sustainability of the practice is ensured both by the spill-over effect of the international workshops as well as the cooperation established between research institutions and researchers. The initiative is scalable and replicable in other countries in the Global South, because the ICGEB has a strong scientific network worldwide, and its Programme management structure has been working effectively for decades. The model of ICGEB's standard procedures and infrastructure, which have a reputation for neutrality, integrity and transparency, are easily reproduced.