RESEARCH PORTFOLIO

WHAT IS GENE THERAPY?

 

I am a scientist, and as you would expect from the words above, my main research efforts over the last two decades have been directed at this fascinating and challenging field. Generally speaking, GENE THERAPY can be defined as a set of strategies used in the field of biomedical research to repair the direct cause of genetic diseases or influence the course of various acquired and multi-factorial disorder. Each approach involves the delivery of a specific nucleic acid (DNA or RNA) with the goal to modify the expression of an individual’s gene or repair abnormal genes. For many diseases, the approach is applied in combination with CELL THERAPY. This is another field of biomedical research that targets various diseases at the cellular level, either by exploiting cells as carriers of therapeutic cargo or by restoring a specific cell population.  

My wishes with this site is to share published and unpublished research studies, like many other scientists, physicians and other professionals have done previously,  to hopefully advance knowledge, education and inspire other with new idea for the development of new therapeutic approaches for the benefit of patients and society. 

 

Cell Biology

The recent advances in post-genomics,  in cellular imaging and systems biology together with standard research methodologies have revolutionized the way with now understand cell biology and their important biological processes, even those acting at the singular cellular component.  This knowledge is of fundamental importance to elaborate and develop new strategy related to the field of non-viral gene therapy or improve existing ones for both ex-vivo and in-vivo therapeutic intervention.         

Gene and Vector Design

In gene therapy,  the approach involves generally the administration of a specific nucleic acid, DNA or RNA. These biomolecules are normally not taken up by cells, thus special carriers, so-called ‘vectors’ are required. Vectors can be of either viral or non-viral nature and their characteristics can be specifically tailored to target a tissue, an organ or even a set of specific cell population. The modifications can be either at the vector self or at the nucleic acid expression systems, which is used to get a gene-products.     

Gene Expression

In four decades, the field of gene therapy has made terrific improvement and also provided reagents, tools, concept and techniques that are now used widely in other field of research as well,  i.e biotechnology,  basic research, microbiology,  environmental science and so on. It is my wish to share with those working in these other fields some unpublished observations and results that I got during my long journey as a scientist working on the improvement of gene-protein expression yield.

WHO I AM

STRATEGY PLANNING

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AAV Molecular model

A very powerful gene therapeutic delivery system

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