2022 has already been a record year for the approval of new gene therapies to treat rare diseases and cancers. More than 2,000 trials are ongoing at the end of June 2022, a slight decrease from the end of 2021. North America leads with about 40%* of active clinical trials, and Europe ranks third with 16%*.
Cell-based immuno-oncology (CAR T cells) represents 34%* of active clinical trials and gene therapies 18%*.
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.
2016-08-04Cell line generation
- Lipid or chemical transfection,
- Viral vectors…
A wide range of tools is available for gene transfer experiments. Among these, the lentiviral vector technology has proven to be an effective tool with significant benefits. This is mainly due to its efficiency, its gentleness with delicate cells, the stability of expression and the shortened time lapse of experiments. Unfortunately, results are not always living up to expectations. To prevent failure, several parameters must be taken into account. We describe in this article 10 best practices to follow for a good transduction efficiency.
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
2016-05-09Cell line generation
Lentiviral vectors insert the sequence of interest right into the host DNA thus allowing for a stable and long-term expression of the transgene. The gene will be copied every time the cell divides.
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!