Over the past decades, the manipulation of genes has led to the development of many methods to generate numerous genetically engineered mouse models for studying human diseases. One major technological leap was achieved by the site-specific recombination technology mediated by the Cre recombinase and its specific DNA “Lox” sites.
At the end of 2018, for the first time in history, the two Chinese twins Lulu and Nana were born with their genome modified by the CRISPR / Cas9 system. This outraged the entire scientific community and ask us about the risks of therapies targeting Human genome. The Cas9 nuclease is often compared to DNA scissors, allowing a very precise cut into any gene. CRISPR are the hands that position these scissors at the desired site of the genome. This system promises to be able to repair any defective gene, thus appearing as the solution for many genetic diseases. It is a valuable tool as well for many therapeutic approaches using genome engineering.
Let’s say you have one or several great gene candidates, you are done with all the in vitro studies and you have been asked to validate your results in vivo. Looking at the bibliography, you inevitably fall into thousands of data showing wonderful results using the Adenoviral Associated Vectors (AAVs). Now you should be asking yourself “what serotype do I need to choose for my project?” and there you go back to bibliography trying to find out a clear answer to this question… and you won’t.
Delivery, delivery, delivery!
Dr Jennifer Doudna opened the last American Society of Gene and Cell Therapy Annual Meeting in Washington by highlighting the current challenges of gene editing :
• Controlling repair pathways
2 different letters and what else?
Everyone uses the word Transfection to designate the deliberate introduction of genetic material (DNA, RNA) into eukaryotic cells. The term is often used whatever the delivery tool but actually it should not!