CRISPR-Cas9 is a really revolutionary technology which was traditionally used to edit genomes. So we repurposed this technology for so-called epigenome editing to really modulate the expression of genes related to Alzheimer’s disease and Parkinson’s disease. So AAV is a gold standard platform for gene delivery, including therapeutic cargo such as CRISPR-Cas9. The main caveat of this technology is that it’s a very small virus, so it can carry only very small cargos or inserts...
CRISPR-Cas9 is a really revolutionary technology which was traditionally used to edit genomes. So we repurposed this technology for so-called epigenome editing to really modulate the expression of genes related to Alzheimer’s disease and Parkinson’s disease. So AAV is a gold standard platform for gene delivery, including therapeutic cargo such as CRISPR-Cas9. The main caveat of this technology is that it’s a very small virus, so it can carry only very small cargos or inserts. So we tackle this problem, and we recently developed this all-in-one adeno-associated vector, or briefly AAV, to carry large and bulky epigenome editing, again, in a single format. The technology is in fairly early stages, but we did a really good job on going into proof of concept for this technology in preclinical settings such as animal settings. So we validated this technology in mice. Specifically, we were able to use it to downregulate expression of APOE gene. So APOE, just for general knowledge, it’s the strongest and more reproducible risk factor for Alzheimer’s disease. So we wanted to downregulate expression of APOE in so-called allele discrimination mode. So we specifically want to downregulate APOE4 without affecting any other isoform such as APOE2 or APOE3 which are neutral or even protective. So this technology based on AAV vector was designed by us and we show a very high efficiency of this technology again in pre-clinical settings such as in animals.
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