A sensible view on gene therapy

https://www.europeanscientist.com/en/public-health/gene-therapy-science-at-a-crossroads/#comment-1782




"Correct or rewrite

Gene therapy is an experimental technique that uses genes to treat or prevent a disease. This incredible odyssey began with the discovery of DNA by Miescher in 1869, the double helix by Watson and Crick in 1953, then continued with the sequencing of the human genome in February 2001, and recently the discovery of CRISPR. Indeed, without excessive simplification, it can be said that living organisms have perfected their genome over the course of their evolution. Consequently, there are often several genes for the same function and conversely several functions for the same gene.

Many techniques can be used to modify the genetic abnormality that causes a disease. It is possible to decrease or switch off the expression of a gene without altering the genetic capital of the individual. To approach it from another angle, you can increase the activity of a gene that supplements the deficient gene. Or you can modify the genetic capital by using a vector that will insert a gene into the genome of cells of various tissues — most often a virus. Finally, it has become possible to rewrite the genetic code, thanks to CRISPR-Cas9, a system derived from bacteria, which suppresses or replaces one gene with another on the DNA at a specific location."

1/ Mitochondria. Mitochondrial donation is special gene therapy which aim is to treat symptomatic genetic anomalies in the DNA of our energy factory.  It is a good but complex example of the issue I raised in this short paper. Shortly mitochondrial donation is a germline therapy so we have to build the predictability models of its long term consequences.
2/ Price. It will be easier than previously. Indeed those techniques rely first on testing the human genome of adults and/or oocytes which is a fully automated process which price is very affordable. Secondly, the lab techniques are far less complex and expensive than doing an open heart operation. So let the market open a free competition between corporations (which is presently the case not only in one country but in all advanced countries) and prices will lower fastly.


1 / Mitochondries. Le don mitochondrial est une thérapie génique spéciale qui vise à traiter les anomalies génétiques symptomatiques dans l'ADN de notre usine d'énergie. C’est un bon exemple, mais complexe, de la question que j’ai soulevée dans ce court document. En bref, le don mitochondrial étant une thérapie germinale, nous devons élaborer des modèles de prévisibilité de ses conséquences à long terme.
2 / Prix. Ce sera plus facile que précédemment. En effet, ces techniques reposent en premier lieu sur le test du génome humain des adultes et / ou des ovocytes, processus entièrement automatisé dont le prix est très abordable. Deuxièmement, les techniques de laboratoire sont beaucoup moins complexes et coûteuses qu’une opération à cœur ouvert. Laissons donc le marché ouvrir la voie à une libre concurrence entre les entreprises (ce qui est actuellement le cas non seulement dans un pays, mais dans tous les pays avancés) et les prix vont baisser rapidement.