This meeting uses to explore new and in-depth developments currently being addressed in RNA therapies.
G. Pavlovic from PHENOMIN-ICS will orally present "The efficiency and simplicity of CRISPR/Cas9 technology to make precise changes to the genome of living cells has led to a new revolution in genetics"
CRISPR/Cas9 genome editing opens new possibilities for modelling human disease in rodents like easy generation of structural variant models and large humanisation of large locus in mouse
Scientists are concerned by CRISPR/Cas9 mediated off-targets but other CRISPR drawbacks are poorly evaluated
Especially, careful characterisation of mouse and rat generated by CRISPR/Cas9 show unexpected mutations like concatemers, ssODN random integration or chromosomal rearrangements
Finally, we will discuss how rodents can be used to evaluate safety of CRISPR/Cas9 therapeutic approaches especially for unexpected genome modifications